Publications

2025

Bensimon G et Al. « "Efficacy and safety of low-dose IL-2 as an add-on therapy to riluzole (MIROCALS): a phase 2b, double-blind, randomised, placebo-controlled trial." » Lancet

Bruge C et Al. « "High-Throughput Screening Identifies Bazedoxifene as a Potential Therapeutic for dysferlin-deficient limb girdle muscular dystrophy" » Br J Pharmacol . 2025 Mar 19

Coppens S et Al. « "Congenital Titinopathy: Comprehensive Characterization of the Most Severe End of the Disease Spectrum." » Annals of Neurology 97(4): 611-628.

Di Leva F et Al. « "SINEUP RNA rescues molecular phenotypes associated with CHD8 suppression in autism spectrum disorder model systems." » Mol Ther 33(3): 1180-1196.

Hirt N et Al. « "Systems immunology integrates the complex endotypes of recessive dystrophic epidermolysis bullosa." » Nature communications 16(1): 664

Laurent M et Al. « "Rescue of lysosomal acid lipase deficiency in mice by rAAV8 liver gene transfer." » Commun Med (Lond) 5(1): 110.

Simoni C et Al. « "Liver fibrosis negatively impacts in vivo gene transfer to murine hepatocytes." » Nature communications 16(1): 2119.

2024

Alarcan H et Al. « Pharmacometabolomics applied to low-dose interleukin-2 treatment in amyotrophic lateral sclerosis » Annals of the New York Academy of Sciences, 10.1111/nyas.15147.

Bullivant J et Al. « The myotubular and centronuclear myopathy patient registry: a multifunctional tool for translational research » Neuromuscular disorders: NMD 35, 42-52.

Clayton JS et Al. « Generation of two iPSC lines from patients with inherited central core disease and concurrent malignant hyperthermia caused by dominant missense variants in the RYR1 gene » Stem cell research 77, 103410.

Clayton JS et Al. « Generation of two iPSC lines from adult central core disease patients with dominant missense variants in the RYR1 gene » Stem cell research 77, 103411.

Clayton JS et Al. « Generation of iPSC lines from three Laing distal myopathy patients with a recurrent MYH7 p.Lys1617del variant » Stem cell research 80, 103491.

Das S et Al. « An empowered, clinically viable hematopoietic stem cell gene therapy for the treatment of multisystemic mucopolysaccharidosis type II » Mol Ther 32, 619-636.

de Feraudy Y et Al. « Exome sequencing in undiagnosed congenital myopathy reveals new genes and refines genes–phenotypes correlations » Genome medicine 16, 87.

Derome M et Al. « [Therapeutic perspectives for lysosomal storage disorders caused by acid ceramidase deficiency] » Med Sci(Paris) 2024 Nov:40 Hors série n° 1:52-55.

Gardin A et Al. « A functional mini-GDE transgene corrects impairment in models of glycogen storage disease type III » The Journal of clinical investigation 134(2):e172018.

Granados A et Al. « SETDB1 modulates the TGFβ response in Duchenne muscular dystrophy myotubes » Science advances 10, eadj8042.

Jaber A et Al. « [Advances and Challenges in Microdystrophin gene therapy for Duchenne Muscular Dystrophy: progress and future directions] » Med Sci (Paris) . 2024 Nov:40 Hors série n° 1:46-51.

Jauze L et Al. « Synergism of dual AAV gene therapy and rapamycin rescues GSDIII phenotype in muscle and liver » JCI insight 9, 10.1172/jci.insight.172614.

La Bella T et Al. « Predictive power of deleterious single amino acid changes to infer on AAV2 and AAV2-13 capsids fitness » Mol Ther Methods Clin Dev . 2024 Aug 20;32(3):101327.

Laugel V et Al. « GNT0004, Genethon's AAV8 vector-delivered microdystrophin gene therapy of Duchenne muscular dystrophy, first data of the phase I/II part of the GNT-016-MDYF all-in-one clinical trial in ambulant boys » Neuromuscular Disorders 43, 410

Lemoine J et Al. « Correction of exon 2, exon 2-9 and exons 8-9 duplications in DMD patient myogenic cells by a single CRISPR/Cas9 system » Scientific reports 14, 21238

Munoz S et Al. « Treatment of infantile-onset Pompe disease in a rat model with muscle-directed AAV gene therapy »

Palmieri L et Al. « In Silico Structural Prediction for the Generation of Novel Performant Midi-Dystrophins Based on Intein-Mediated Dual AAV Approach » Int J Mol Sci . 2024 Sep 27;25(19):10444.

Pulman J et Al. « Direct delivery of Cas9 or base editor protein and guide RNA complex enables genome editing in the retina » Mol Ther Nucleic Acids . 2024 Sep 30;35(4):102349.

Raby A et Al. « Spastin regulates ER-mitochondrial contact sites and mitochondrial homeostasis » iScience 27, 11068

Sellier S et Al. « Muscle-specific, liver-detargeted adeno-associated virus gene therapy rescues Pompe phenotype in adult and neonate Gaa−/− mice » Journal of inherited metabolic disease 47, 119-134.

Shi x et Al. « Repeated dosing of AAV-mediated liver gene therapy in juvenile rat and mouse models of Crigler-Najjar syndrome type I » Mol Ther Methods Clin Dev . 2024 Oct 28;32(4):101363.

Vendomele J et Al. « Peripheral Cellular Immune Responses Induced by Subretinal Adeno-Associated Virus Gene Transfer Can Be Restrained by the Subretinal-Associated Immune Inhibition Mechanism » Human gene therapy 35, 464-476.

Vu Hong A et Al. « An engineered AAV targeting integrin alpha V beta 6 presents improved myotropism across species » Nature communications 15, 7965

2023

Albini S et Al. « Assessment of Therapeutic Potential of a Dual AAV Approach for Duchenne Muscular Dystrophy » International Journal of Molecular Sciences, 24,11421

Aronson SJ et Al. « What's next in gene therapy for Crigler-Najjar syndrome? » Expert opinion on biological therapy 23, 119-121.

Barbon E et Al. « Development of a dualIP – 11/12/202339hybrid AAV vector for endothelial-targeted expression of von Willebrand factor. » Gene therapy 30, 245-254.

Berling E et Al. « Gene therapy review: Duchenne muscular dystrophy case study » Revue neurologique 179, 90-105

Brusson M et Al. « Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies. » Molecular therapy Nucleic acids 32, 229-246.

Cardone N et Al. « Clinical and functional characterization of a long survivor congenital titinopathy patient with a novel metatranscript-only titin variant » Acta neuropathologica communications 11, 48.

Chatrousse L et Al. « Establishment of heterozygous and homozygous SHANK3 knockout clonal pluripotent stem cells from the parental hESC line SA001 using CRISPR/Cas9 » Stem cell research 72, 103209.

Corre G et Al. « Evaluation of diversity indices to estimate clonal dominance in gene therapy studies » Molecular therapy Methods & clinical development 29, 418-425.

D'Antiga L et Al. « Gene Therapy in Patients with the Crigler-Najjar Syndrome » The New England journal of medicine 389, 620-631.

Dalla Barba F et Al. « Modeling Sarcoglycanopathy in Danio rerio » International journal of molecular sciences 24, 12707

Driver K et Al. « Generation of two induced pluripotent stem cell lines from a 33-year-old central core disease patient with a heterozygous dominant c.14145_14156delCTACTGGGACA (p.Asn4715_Asp4718del) deletion in the RYR1 gene » Stem cell research, Available online 22 November 2023.

Earley J et Al. « Evading and overcoming AAV neutralization in gene therapy » Trends in biotechnology 41, 836-845.

Ferguson LP et Al. « Smarcd3 is an epigenetic modulator of the metabolic landscape in pancreatic ductal adenocarcinoma » Nature communications 14, 292.

Ferla R et Al. « Efficacy, pharmacokinetics, and safety in the mouse and primate retina of dual AAV vectors forIP – 11/12/202340Usher syndrome type 1B » Molecular therapy Methods & clinical development 28, 396-411.

Gardin A et AL. « A functional mini-GDE transgene corrects impairment in models of glycogen storage disease type III » The Journal of clinical investigation.

Gardin A et AL. « Lessons learned from clinical experience with AAV vectors. » Archives de pediatrie : organe officiel de la Societe francaise de pediatrie 30, 8S46-48S52.

Geoffroy M et Al. « CRISPR-Cas9 KO Cell Line Generation and Development of a Cell-Based Potency Assay for rAAV-FKRP Gene Therapy » Gene Therapy. Cells 12, 2444.

Labrosse R et Al. « Outcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome » Blood 142, 1281-1296.

Lisjak M et Al. « Lethality rescue and long-term amelioration of a citrullinemia type I mouse model by neonatal gene-targeting combined to SaCRISPR-Cas9. » Molecular therapy Methods & clinical development 31, 101103.

Morin A et Al. « Dystrophin myonuclear domain restoration governs treatment efficacy in dystrophic muscle » Proc Natl Acad Sci U S A. 2023 Jan 3;120(2):e2206324120.

Piletska E et Al. « Analysis of Adeno-Associated Virus Serotype 8 (AAV8)-antibody complexes using epitope mapping by molecular imprinting leads to the identification of Fab peptides that potentially evade AAV8 neutralisation » Nanomedicine 52, 102691.

Prakasam R et Al. « LSD1/PRMT6-targeting gene therapy to attenuate androgen receptor toxic gain-of-function ameliorates spinobulbar muscular atrophy phenotypes in flies and mice » Nature communications 14, 603

Richard I « Basic notions about gene therapy from the nucleic acid perspective and applications in a pediatric disease: Duchenne muscular dystrophy. » Archives de pediatrie : organe officiel de la Societe francaise de pediatrie 30, 8S2-8S11.

Rossiaud L et Al. « Generation of three induced pluripotent stem cell lines from patients with glycogen storage disease type III » Stem cell research 72, 103214.

Rossiaud L et Al. « Pathological modeling of glycogen storage disease type III with CRISPR/Cas9 edited human pluripotent stem cells » Frontiers in cell and developmental biology 11, 1163427.

Sellier P et Al. « Muscle-specific, liver-detargeted adeno-associated virus gene therapy rescues Pompe phenotype in adult and neonate Gaa(-/-) mice » Journal of inherited metabolic disease.

Shieh PB et Al. « Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial » Lancet neurology 22, 1125-1139. 26.

Steicy Sobrino et Al « Severe hematopoeitic stem cell inflammation compromises chronic granulomatous disease gene therapy » Cell Reports Medicine

Szentesi P et Al. « Disrupted T-tubular network accounts for asynchronous calcium release in MTM1-deficient skeletal muscle » The Journal of physiology 601, 99-121

Vu Hong A et Al « Dlk1-Dio3 cluster miRNAs regulate mitochondrial functions in the dystrophic muscle in Duchenne muscular dystrophy » Life science alliance 6, e202201506

2022

Ail D. et Al. « Systemic and local immune responses to intraocular AAV vector administration in non-human primates » Molecular therapy Methods & clinical development 24, 306-316

Brunetti-Pierri N et Al. « Liver-Directed Adeno-Associated Virus–Mediated<br>Gene Therapy for Mucopolysaccharidosis Type VI » NEJM Evidence, 1-12

A. Magnani et Al « Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome » Nature Medecine 28, 71-80

Aguilar-Gonzalez A. et Al. « Isogenic GAA-KO Murine Muscle Cell Lines Mimicking Severe Pompe Mutations as Preclinical Models for the Screening of Potential Gene Therapy Strategies » International journal of molecular sciences 11, 4-23

Amendola M. et Al. « CRISPRthripsis: “The Risk of CRISPR/Cas9-induced Chromothripsis in Gene Therapy » Stem cells translational medicine 11, 1003-1009

Antoniou P. et Al. « Base-editing-mediated dissection of a gamma-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression » Nature communications 13, 6618.

Aronson SJ et Al. « What's next in gene therapy for Crigler-Najjar syndrome? » Expert opinion on biological therapy, 1-3.

Bourg N.et Al. « Co-Administration of Simvastatin Does Not Potentiate the Benefit of Gene Therapy in the mdx Mouse Model for Duchenne Muscular Dystrophy » International journal of molecular sciences 23, 2016.

Bruge C. et Al. « Skeletal Muscle Cells Derived from Induced Pluripotent Stem Cells: A Platform for Limb Girdle Muscular Dystrophies » Biomedicines 10, 1428..

Buffa V et Al. « Hematopoietic stem and progenitors cells gene editing: Beyond blood disorders » Front Genome Ed 4, 997142

Clayton JS et Al. « Generation of an induced pluripotent stem cell line from a 3-month-old nemaline myopathy patient with a heterozygous dominant c.515C > A (p.Ala172Glu) variant in the ACTA1 gene » Stem cell research 63, 102829

Corre G. et Al. « Lentiviral standards to determine the sensitivity of assays that quantify lentiviral vector copy numbers and genomic insertion sites in cells » Gene therapy 29, 536-543.

El Andari J. et Al. « Semirational bioengineering of AAV vectors with increased potency and specificity for systemic gene therapy of muscle disorders » Science advances 8, eabn4704.

Errera MH. et Al. « Cytokines, chemokines and growth factors profile in human aqueous humor in idiopathic uveitis » PloS one 17, e0254972.

Gross DA. et Al. « Overcoming the Challenges Imposed by Humoral Immunity to AAV Vectors to Achieve Safe and Efficient Gene Transfer in Seropositive Patients » Frontiers in immunology 13, 857276.

Hoch L. et Al. « Dual Blockade of Misfolded Alpha-Sarcoglycan Degradation by Bortezomib and Givinostat Combination » Frontiers in pharmacology 13, 856804.

Israeli D. et Al. « Deciphering the Molecular Mechanism of Incurable Muscle Disease by a Novel Method for the Interpretation of miRNA Dysregulation » Non-coding RNA 8, 48.

Labasse C. et Al. « Severe ACTA1-related nemaline myopathy: intranuclear rods, cytoplasmic bodies, and enlarged perinuclear space as characteristic pathological features on muscle biopsies » Acta neuropathologica communications 10, 101.

Magnani A et Al. « Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott-Aldrich syndrome » Nat Med 28, 71-80

Parmentier R et Al. « Global genome decompaction leads to stochastic activation of gene expression as a first step toward fate commitment in human hematopoietic cells » PLoS biology 20, e3001849

Ramadier S. et Al. « Combination of lentiviral and genome editing technologies for the treatment of sickle cell disease » Mol Ther 30, 145-163.

Reinbigler M et Al. « Artificial intelligence workflow quantifying muscle features on Hematoxylin-Eosin stained sections reveals dystrophic phenotype amelioration upon treatment » Scientific reports 12, 19913

Richard I. et Al. « Le registre national des calpaïnopathies » Les cahiers de myologie (EDP Sciences), pp. 33-34.

Romano G. et Al. « Rescue of a familial dysautonomia mouse model by AAV9-Exon-specific U1 snRNA » American journal of human genetics 109, 1534-1548.

Suleski IS et Al. « Generation of two isogenic induced pluripotent stem cell lines from a 1-month-old nemaline myopathy patient harbouring a homozygous recessive c.121C > T (p.Arg39Ter) variant in the ACTA1 gene » Stem cell research 63, 102830

Teyssou E. et Al. « The Amyotrophic Lateral Sclerosis M114T PFN1 Mutation Deregulates Alternative Autophagy Pathways and Mitochondrial Homeostasis » International journal of molecular sciences 23, 5694.

Trubetskoy V. et Al « Mapping genomic loci implicates genes and synaptic biology in schizophrenia » Nature 604, 502-508.

Vecten M. et Al. « Objective evaluation of clinical actionability for genes involved in myopathies: 63 genes with a medical value for patient care » International journal of molecular sciences 23, 8506.

Zabaleta N. et Al. « Durable immunogenicity, adaptation to emerging variants, and low-dose efficacy of an AAV-based COVID-19 vaccine platform in macaques » Mol Ther 30, 2952-2967.

2021

Amendola M. et Al. « Recent progress in genome editing for gene therapy applications: the French perspective » Human gene therapy

Amor, F. et Al. « Cholesterol metabolism is a potential therapeutic target in Duchenne muscular dystrophy » Journal of cachexia, sarcopenia and muscle 12, 677-693

Andrieu, M. et Al. « La biosécurité et la cytométrie en flux. In La cytométrie en flux – 2ème édition » L. Tec&Doc, ed., Chapter 29, pp. 483-492

Barbon, E. et Al. « Development of a dual hybrid AAV vector for endothelial-targeted expression of von Willebrand factor » Gene therapy

Berling, E. et Al. « Narrative review of glycogen storage disorder type III with a focus on neuromuscular, cardiac and therapeutic aspects » Journal of inherited metabolic disease 44, 521-533

Biquand, A. et Al. « Titin M-line insertion sequence 7 is required for proper cardiac function in mice » Journal of cell science 134

Chan, YK. et Al. « Engineering adeno-associated viral vectors to evade innate immune and inflammatory responses » Science translational medicine 13

Chandra, G. et Al. « Endoplasmic reticulum maintains ion homeostasis required for plasma membrane repair » The Journal of cell biology 220 (5)

Clayton, JS et Al. « Generation of two isogenic induced pluripotent stem cell lines from a 4-month-old severe nemaline myopathy patient with a heterozygous dominant c.553C > A (p.Arg183Ser) variant in the ACTA1 gene » Stem cell research 53, 102273

Clayton, JS et Al. « Generation of two isogenic induced pluripotent stem cell lines from a 10-year-old typical nemaline myopathy patient with a heterozygous dominant c.541G>A (p.Asp179Asn) pathogenic variant in the ACTA1 gene » Stem cell research 55, 102482

Costa-Verdera, H. « Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates » Nature communications 12, 6393

De Sabbata, G, et Al. « Long-term correction of ornithine transcarbamylase deficiency in Spf-Ash mice with a translationally optimized AAV vector » Molecular therapy Methods & clinical development 20, 169-180

Delville, M. et Al. « A combination of cyclophosphamide and interleukin-2 allows CD4+ T cells converted to Tregs to control scurfy syndrome » Blood 137, 2326-2336

Fouarge, E. et Al. « Hierarchical Bayesian modelling of disease progression to inform clinical trial design in centronuclear myopathy » Orphanet journal of rare diseases 16 (1), 3

Galibert, L. et Al. « Monobac System-A Single Baculovirus for the Production of rAAV » Microorganisms 9

Giovannelli, I. et Al. « Amyotrophic lateral sclerosis transcriptomics reveals immunological effects of low-dose interleukin-2 » Brain communications 3, fcab141

Guimaraes-Costa R F-EG. et Al. « Clinical correlations and long-term follow-up in 100 patients with sarcoglycanopathies » European journal of neurology 28, 660-669

Long BR. et Al. « Early Phase Clinical Immunogenicity of Valoctocogene Roxaparvovec, an AAV5-Mediated Gene Therapy for Hemophilia A » Mol Ther 29, 597-610

Lornage, X. et Al. « Selective loss of a LAP1 isoform causes a muscle-specific nuclear envelopathy » Neurogenetics 22, 33-41

Marinello, M. « Characterization of Neuromuscular Junctions in Mice by Combined Confocal and Super-Resolution Microscopy » Journal of visualized experiments : JoVE 178

Morciano, G. et Al. « A naturally occurring mutation in ATP synthase subunit c is associated with increased damage following hypoxia/reoxygenation in STEMI patients » Cell reports 35, 108983

Parrini, M. et Al. « Restoring neuronal chloride homeostasis with anti-NKCC1 gene therapy rescues cognitive deficits in a mouse model of Down syndrome » Mol Ther 6 ;29(10), 3072-3092

Pavani, G. et Al. « Targeted gene delivery: Where to land » Frontiers in Genome Editing, volume 2, Open Access

Pavani, G. et Al. « Correction of beta-thalassemia by CRISPR/Cas9 editing of the alpha-globin locus in human hematopoietic stem cells » Blood advances 5, 1137-1153

Ramadier, S. et Al. « Combination of lentiviral and genome editing technologies for the treatment of sickle cell disease » Mol Ther

Rees, M. et Al. « Making sense of missense variants in TTN-related congenital myopathies » Acta neuropathologica 141, 431-453

Shi X. et Al. « Efficacy of AAV8-hUGT1A1 with Rapamycin in neonatal, suckling, and juvenile rats to model treatment in pediatric CNs patients » Molecular therapy Methods & clinical development 20, 287-297

Spinazzi, M. et Al. « Late-onset camptocormia caused by a heterozygous in-frame CAPN3 deletion » Neuromuscular disorders : NMD 31, 450-455

Spinozzi, S. « Calpains for dummies: What you need to know about the calpain family » Biochimica et biophysica acta Proteins and proteomics 1869, 140616

Teyssou, E. et Al. « Genetic screening of ANXA11 revealed novel mutations linked to amyotrophic lateral sclerosis » Neurobiology of aging 99, 102 e111-102 e120

Thiruvengadam, G. et Al. « Anoctamin 5 Knockout Mouse Model Recapitulates LGMD2L Muscle Pathology and Offers Insight Into in vivo Functional Deficits » J Neuromuscul Dis 8, S243-S255

Vaubourg, C. et Al. « Minimal Consequences of CMAH and DBA/2 Backgrounds on a FKRP Deficient Model » J Neuromuscul Dis 8, 785-793

Vu Hong, A. et Al. « A revised model for mitochondrial dysfunction in Duchenne muscular dystrophy » Eur J Transl Myol 3

Zhukouskaya, VV. et Al. « A novel therapeutic strategy for skeletal disorders: Proof of concept of gene therapy for X-linked hypophosphatemia » Science advances 7, eabj5018

2020

Barbon E., et al. (2020) « Single-domain antibodies targeting antithrombin reduce bleeding in hemophilic mice with or without inhibitors » EMBO molecular medicine 12, e11298.

Bertin B., et al. (2020) « Capsid-specific removal of circulating antibodies to adeno-associated virus vectors » Scientific reports 10, 864.

Breuer CB., et al (2020) « In vivo engineering of lymphocytes after systemic exosome-associated AAV delivery » Scientific reports 10, 4544.

Cagin U., et al. (2020) « Rescue of Advanced Pompe Disease in Mice with Hepatic Expression of Secretable Acid alpha-Glucosidase » Mol Ther.

Carotti M., et al. (2020) « Combined Use of CFTR Correctors in LGMD2D Myotubes Improves Sarcoglycan Complex Recovery » Int J Mol Sci 21(5), 1813.

Dupont JB., et al. (2020) « AAV-Mediated Gene Transfer Restores a Normal Muscle Transcriptome in a Canine Model of X-Linked Myotubular Myopathy » Mol Ther 28(2), 382-393.

Espinoza S., et al. (2020) « SINEUP Non-coding RNA Targeting GDNF Rescues Motor Deficits and Neurodegeneration in a Mouse Model of Parkinson's Disease » Mol Ther 28(2), 642-652.

Jmii H., et al. (2020) « Immunopathology in the brain of mice following vertical transmission of Coxsackievirus B4 » Microb Pathog 140, 103965.

Jmii H., et al. (2020) « Coxsackievirus B4 infection and interneuronal spread in primary cultured neurons » Microb Pathog 145, 104235.

Kohn DB., et al. (2020) « Lentiviral gene therapy for X-linked chronic granulomatous disease » Nat Med 26(2), 200-206.

Leborgne C., et al. (2020) « IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies » Nat Med 26(7), 1096-1101.

Marquette A., et al. (2020) « Peptides derived from the C-terminal domain of HIV-1 Viral Protein R in lipid bilayers: Structure, membrane positioning and gene delivery » Biochim Biophys Acta Biomembr 1862(2), 183149.

Pavani G., et al. (2020) « Ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins » Nat Commun 11(1), 3778.

Perrin A., et al. (2020) « A new congenital multicore titinopathy associated with fast myosin heavy chain deficiency » Ann Clin Transl Neurol 7(5), 846-854.

Romano O., et al. (2020) « GATA Factor-Mediated Gene Regulation in Human Erythropoiesis » iScience 23(4), 101018.

Ronzitti G., et al. (2020) « Human Immune Responses to Adeno-Associated Virus (AAV) Vectors » Front Immunol 11, 670.

Six E., et al. (2020) « Clonal tracking in gene therapy patients reveals a diversity of human hematopoietic differentiation programs » Blood 135(15), 1219-1231.

Taheri H., et al. (2020) « Photocatalytically Active Graphitic Carbon Nitride as an Effective and Safe 2D Material for In Vitro and In Vivo Photodynamic Therapy » Small 16(10), e1904619.

Telailia N., et al. (2020) « Compression of The Sciatic Nerve May not Contribute to Ipsilateral Hyperalgesia Development in Ovariectomized Female Rats! » Cell J 22(4), 548-555.

Verdera HC., et al. (2020) « AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer » Mol Ther 28(3), 723-746.

Vissing J., et al. (2020) « A single c.1715G>C calpain 3 gene variant causes dominant calpainopathy with loss of calpain 3 expression and activity » Hum Mutat 41(9), 1507-1513.

Weber L., et al. (2020) « Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype » Sci Adv 6(7), eaay9392.

2019

Annoussamy, M., et al. (2019). « X-linked myotubular myopathy: A prospective international natural history study. » Neurology 92(16): e1852-e1867.

Aronson, S. J., et al. (2019). « Liver-directed gene therapy results in long-term correction of progressive familial intrahepatic cholestasis type 3 in mice. » J Hepatol 71(1): 153-162.

Aronson, S. J., et al. (2019). « Prevalence and Relevance of Pre-Existing Anti-Adeno-Associated Virus Immunity in the Context of Gene Therapy for Crigler-Najjar Syndrome. » Hum Gene Ther 30(10): 1297-1305.

Bartolo, L., et al. (2019). « Dual muscle-liver transduction imposes immune tolerance for muscle transgene engraftment despite preexisting immunity. » JCI Insight 4(11).

Boudeffa, D., et al. (2019). « Towards a scalable purification protocol of GaLV-TR pseudotyped lentiviral vectors. » Hum Gene Ther Methods.

Buning, H., et al. (2019). « Breaking the Barriers of Genetic and Metabolic Disorders. » Hum Gene Ther 30(10): 1177-1179.

Casajus Pelegay, E., et al. (2019). « Targeting Mitochondrial Defects to Increase Longevity in Animal Models of Neurodegenerative Diseases. » Adv Exp Med Biol 1134: 89-110.

Charrier, S., et al. (2019). « Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID. » Mol Ther Methods Clin Dev 15: 232-245.

Colella, P. and F. Mingozzi (2019). « Gene Therapy for Pompe Disease: The Time is now. » Hum Gene Ther 30(10): 1245-1262.

Colella, P., et al. (2019). « AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice. » Mol Ther Methods Clin Dev 12: 85-101.

Collaud, F., et al. (2019). « Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome » Mol Ther Methods Clin Dev 12: 157-174.

Da Rocha, S., et al. (2019). « Temporary Reduction of Membrane CD4 with the Antioxidant MnTBAP Is Sufficient to Prevent Immune Responses Induced by Gene Transfer. » Mol Ther Methods Clin Dev 14: 285-299.

Dupont, J. B., et al. (2019). « AAV-Mediated Gene Transfer Restores a Normal Muscle Transcriptome in a Canine Model of X-Linked Myotubular Myopathy. » Mol Ther.

Espinoza, S., et al. (2019). « SINEUP Non-coding RNA Targeting GDNF Rescues Motor Deficits and Neurodegeneration in a Mouse Model of Parkinson's Disease. » Mol Ther.

Gross, D. A., et al. (2019). « Induction of tumor-specific CTL responses using the C-terminal fragment of Viral protein R as cell penetrating peptide. » Sci Rep 9(1): 3937.

Henriques, S. F., et al. (2019). « Functional and cellular localization diversity associated with Fukutin-related protein patient genetic variants. » Hum Mutat 40(10): 1874-1885.

Hoch, L., et al. (2019). « Identification of thiostrepton as a pharmacological approach to rescue misfolded alpha-sarcoglycan mutant proteins from degradation. » Sci Rep 9(1): 6915.

Israeli, D., et al. (2019). « An AAV-SGCG Dose-Response Study in a gamma-Sarcoglycanopathy Mouse Model in the Context of Mechanical Stress. » Mol Ther Methods Clin Dev 13: 494-502.

Jauze, L., et al. (2019). « Challenges of Gene Therapy for the Treatment of Glycogen Storage Diseases Type I and Type III. » Hum Gene Ther 30(10): 1263-1273.

Kutchukian, C., et al. (2019). « Ca(2+)-induced sarcoplasmic reticulum Ca(2+) release in myotubularin-deficient muscle fibers. » Cell Calcium 80: 91-100.

Laforet, P., et al. (2019). « Deep morphological analysis of muscle biopsies from type III glycogenesis (GSDIII), debranching enzyme deficiency, revealed stereotyped vacuolar myopathy and autophagy impairment. » Acta Neuropathol Commun 7(1): 167.

Lattanzi, A., et al. (2019). « Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements. » Mol Ther 27(1): 137-150.

Le Quellec, S., et al. (2019). « Recombinant Adeno-Associated Viral Vectors Expressing Human Coagulation FIX-E456H Variant in Hemophilia B Mice. » Thromb Haemost 119(12): 1956-1967.

Leborgne, C., et al. (2019). « Prevalence and long-term monitoring of humoral immunity against adeno-associated virus in Duchenne Muscular Dystrophy patients. » Cell Immunol 342: 103780.

Lo Scrudato, M., et al. (2019). « Genome Editing of Expanded CTG Repeats within the Human DMPK Gene Reduces Nuclear RNA Foci in the Muscle of DM1 Mice. » Mol Ther 27(8): 1372-1388.

Lostal, W., et al. (2019). " « In Vivo Monitoring of Calpain Activity by Forster Resonance Energy Transfer » Methods Mol Biol 1915: 57-66.

Moussy, A., et al. (2019). « Constraints on Human CD34+ Cell Fate due to Lentiviral Vectors Can Be Relieved by Valproic Acid. » Hum Gene Ther.

Orefice, N. S., et al. (2019). « Real-Time Monitoring of Exosome Enveloped-AAV Spreading by Endomicroscopy Approach: A New Tool for Gene Delivery in the Brain. » Mol Ther Methods Clin Dev 14: 237-251.

Pignani, S., et al. (2019). « Tailoring the CRISPR system to transactivate coagulation gene promoters in normal and mutated contexts. » Biochim Biophys Acta Gene Regul Mech 1862(6): 619-624.

Poupiot, J., et al. (2019). « Role of Regulatory T Cell and Effector T Cell Exhaustion in Liver-Mediated Transgene Tolerance in Muscle. » Mol Ther Methods Clin Dev 15: 83-100.

Radek, C., et al. (2019). « Vectofusin-1 improves transduction of primary human cells with diverse retroviral and lentiviral pseudotypes, enabling robust, automated closed-system manufacturing. » Hum Gene Ther.

Ronzitti, G., et al. (2019). « Progress and challenges of gene therapy for Pompe disease. » Ann Transl Med 7(13): 287.

Scott, D. W. and F. Mingozzi (2019). « Balancing immunity and tolerance in gene therapy for inherited and acquired diseases. » Cell Immunol 342: 103945.

2018

Antoniani, C., et al. (2018). « Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human beta-globin locus. » Blood 131(17): 1960-1973.

Avila-Polo, R., et al. (2018). « Loss of Sarcomeric Scaffolding as a Common Baseline Histopathologic Lesion in Titin-Related Myopathies. » J Neuropathol Exp Neurol 77(12): 1101-1114.

Brendel, C., et al. (2018). « Non-Clinical Efficacy and Safety Studies on G1XCGD, a Lentiviral Vector for Ex Vivo Gene Therapy of X-Linked Chronic Granulomatous Disease. » Hum Gene Ther Clin Dev 29(2): 69-79.

Carotti, M., et al. (2018). « Repairing folding-defective alpha-sarcoglycan mutants by CFTR correctors, a potential therapy for Limb Girdle Muscular Dystrophy 2D. » Hum Mol Genet.

Colella, P., et al. (2018). « Emerging Issues in AAV-Mediated In Vivo Gene Therapy. » Mol Ther Methods Clin Dev 8: 87-104.

Daniele, N., et al. (2018). « Intravenous Administration of a MTMR2-Encoding AAV Vector Ameliorates the Phenotype of Myotubular Myopathy in Mice. » J Neuropathol Exp Neurol 77(4): 282-295.

Denard, J., et al. (2018). « AAV-8 and AAV-9 Vectors Cooperate with Serum Proteins Differently Than AAV-1 and AAV-6. » Mol Ther Methods Clin Dev 10: 291-302.

Domenger, C., et al. (2018). « RNA-Seq Analysis of an Antisense Sequence Optimized for Exon Skipping in Duchenne Patients Reveals No Off-Target Effect. » Mol Ther Nucleic Acids 10: 277-291.

Donadon, I., et al. (2018). « Exon-specific U1 snRNAs improve ELP1 exon 20 definition and rescue ELP1 protein expression in a familial dysautonomia mouse model. » Hum Mol Genet 27(14): 2466-2476.

Fitzpatrick, Z., et al. (2018). « Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction. » Mol Ther Methods Clin Dev 9: 119-129.

Galibert, L., et al. (2018). « Origins of truncated supplementary capsid proteins in rAAV8 vectors produced with the baculovirus system. » PLoS One 13(11): e0207414.

Henriques, S. F., et al. (2018). « Different outcome of sarcoglycan missense mutation between human and mouse. » PLoS One 13(1): e0191274.

Holstein, M., et al. (2018). « Efficient Non-viral Gene Delivery into Human Hematopoietic Stem Cells by Minicircle Sleeping Beauty Transposon Vectors. » Mol Ther 26(4): 1137-1153.

Izmiryan, A., et al. (2018). « Ex Vivo COL7A1 Correction for Recessive Dystrophic Epidermolysis Bullosa Using CRISPR/Cas9 and Homology-Directed Repair. » Mol Ther Nucleic Acids 12: 554-567.

Jonson, P. H., et al. (2018). « Novel mutations in DNAJB6 cause LGMD1D and distal myopathy in French families. » Eur J Neurol 25(5): 790-794.

Khabou, H., et al. (2018). « Dosage Thresholds and Influence of Transgene Cassette in Adeno-Associated Virus-Related Toxicity. » Hum Gene Ther 29(11): 1235-1241.

Kuranda, K., et al. (2018). « Exposure to wild-type AAV drives distinct capsid immunity profiles in humans. » J Clin Invest 128(12): 5267-5279.

Lidonnici, M. R., et al. (2018). « Multiple Integrated Non-clinical Studies Predict the Safety of Lentivirus-Mediated Gene Therapy for beta-Thalassemia. » Mol Ther Methods Clin Dev 11: 9-28.

Lostal, W., et al. (2018). « 233rd ENMC International Workshop: Clinical Trial Readiness for Calpainopathies, Naarden, The Netherlands, 15-17 September 2017. » Neuromuscul Disord 28(6): 540-549.

Meliani, A., et al. (2018). « Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration. » Nat Commun 9(1): 4098.

Mingozzi, F. (2018). « AAV Immunogenicity: A Matter of Sensitivity. » Mol Ther 26(10): 2335-2336.

Oates, E. C., et al. (2018). « Congenital Titinopathy: Comprehensive characterization and pathogenic insights. » Ann Neurol 83(6): 1105-1124.

Paldi, A. (2018). « Conceptual Challenges of the Systemic Approach in Understanding Cell Differentiation. » Methods Mol Biol, Springer. 1702: 27-39.

Paulk, N. K., et al. (2018). « Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity. » Mol Ther 26(1): 289-303.

Poletti, V. and F. Mavilio (2018). « Interactions between Retroviruses and the Host Cell Genome. » Mol Ther Methods Clin Dev 8: 31-41.

Poletti, V., et al. (2018). « Preclinical Development of a Lentiviral Vector for Gene Therapy of X-Linked Severe Combined Immunodeficiency. » Mol Ther Methods Clin Dev 9: 257-269.

Poletti, V., et al. (2018). « Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling βAS3 Globin for Gene Therapy for Sickle Cell Disease. » Mol Ther Methods Clin Dev 11: 167-179.

Ronzitti, G. and F. Mingozzi (2018). « Combination Therapy Is the New Gene Therapy? » Mol Ther 26(1): 12-14.

Rouillon, J., et al. (2018). « High urinary ferritin reflects myoglobin iron evacuation in DMD patients. » Neuromuscul Disord 28(7): 564-571.

Urbinati, F., et al. (2018). « Gene Therapy for Sickle Cell Disease: A Lentiviral Vector Comparison Study. » Hum Gene Ther 29(10): 1153-1166.

Vendomele, J., et al. (2018). « Subretinal Injection of HY Peptides Induces Systemic Antigen-Specific Inhibition of Effector CD4(+) and CD8(+) T-Cell Responses. » Front Immunol 9: 504.

Vidal, P., et al. (2018). « Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression. » Mol Ther 26(3): 890-901.

Weber, L., et al. (2018). « An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype. » Mol Ther Methods Clin Dev 10: 268-280.

Weihl, C. C., et al. (2018). « 234th ENMC International Workshop: Chaperone dysfunction in muscle disease Naarden, The Netherlands, 8-10 December 2017. » Neuromuscul Disord 28(12): 1022-1030.

2017

Boucheham, A., et al. (2017). « IpiRId: Integrative approach for piRNA prediction using genomic and epigenomic data. » PLoS One 12(6): e0179787.

Cosette, J., et al. (2017). « Combination of imaging flow cytometry and time-lapse microscopy for the study of label-free morphology dynamics of hematopoietic cells. » Cytometry A 91(3): 254-260.

Dalichaouche, I., et al. (2017). « Gamma-sarcoglycan and dystrophin mutation spectrum in an Algerian cohort. » Muscle Nerve 56(1): 129-135.

Elverman, M., et al. (2017). « Long-term effects of systemic gene therapy in a canine model of myotubular myopathy. » Muscle Nerve 56(5): 943-953.

Galy, A. (2017). « Major Advances in the Development of Vectors for Clinical Gene Therapy of Hematopoietic Stem Cells from European Groups over the Last 25 Years. » Hum Gene Ther 28(11): 964-971.

Gicquel, E., et al. (2017). « AAV-mediated transfer of FKRP shows therapeutic efficacy in a murine model but requires control of gene expression. » Hum Mol Genet 26(10): 1952-1965.

Han, S. O., et al. (2017). « Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance Induction. » Mol Ther Methods Clin Dev 4: 126-136.

Hardet, R. and F. Mingozzi (2017). « Oral Tolerance: Another Reason to Eat Your Veggies! » Mol Ther 25(2): 311-313.

Holic, N., et al. (2017). « Improvement of De Novo Cholesterol Biosynthesis Efficiently Promotes the Production of Human Immunodeficiency Virus Type 1-Derived Lentiviral Vectors. » Hum Gene Ther Methods 28(2): 67-77.

Hosel, M., et al. (2017). « Autophagy determines efficiency of liver-directed gene therapy with adeno-associated viral vectors. » Hepatology 66(1): 252-265.

Le Guiner, C., et al. (2017). « Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy. » Nat Commun 8: 16105.

Leborgne, C., et al. (2017). « The absorption enhancer sodium deoxycholate promotes high gene transfer in skeletal muscles. » Int J Pharm 523(1): 291-299.

Mack, D. L., et al. (2017). « Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs. » Mol Ther 25(4): 839-854.

Majdoul, S., et al. (2017). « Peptides derived from evolutionarily conserved domains in Beclin-1 and Beclin-2 enhance the entry of lentiviral vectors into human cells. » J Biol Chem 292(45): 18672-18681.

Mariot, V., et al. (2017). « Downregulation of myostatin pathway in neuromuscular diseases may explain challenges of anti-myostatin therapeutic approaches. » Nat Commun 8(1): 1859.

Marsolier, J., et al. (2017). « 1st International Workshop on Clinical trial readiness for sarcoglycanopathies 15-16 November 2016, Evry, France. » Neuromuscul Disord 27(7): 683-692.

Matet, A., et al. (2017). « Evaluation of tolerance to lentiviral LV-RPE65 gene therapy vector after subretinal delivery in non-human primates. » Transl Res 188: 40-57 e44.

Mavilio, F. (2017). « Developing gene and cell therapies for rare diseases: an opportunity for synergy between academia and industry. » Gene Ther 24(9): 590-592.

Meliani, A., et al. (2017). « Enhanced liver gene transfer and evasion of preexisting humoral immunity with exosome-enveloped AAV vectors. » Blood Adv 1(23): 2019-2031.

Mingozzi, F. and K. A. High (2017). « Overcoming the Host Immune Response to Adeno-Associated Virus Gene Delivery Vectors: The Race Between Clearance, Tolerance, Neutralization, and Escape. » Annu Rev Virol 4(1): 511-534.

Morris, E. C., et al. (2017). « Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult. » Blood 130(11): 1327-1335.

Moulay, G., et al. (2017). « Histidine-rich designer peptides of the LAH4 family promote cell delivery of a multitude of cargo. » J Pept Sci 23(4): 320-328.

Moussy, A., et al. (2017). « Integrated time-lapse and single-cell transcription studies highlight the variable and dynamic nature of human hematopoietic cell fate commitment. » PLoS Biol 15(7): e2001867.

Nagy, N., et al. (2017). « Hip region muscular dystrophy and emergence of motor deficits in dysferlin-deficient Bla/J mice. » Physiol Rep 5(6).

Piovan, C., et al. (2017). « Vectofusin-1 Promotes RD114-TR-Pseudotyped Lentiviral Vector Transduction of Human HSPCs and T Lymphocytes. » Mol Ther Methods Clin Dev 5: 22-30.

Puzzo, F., et al. (2017). « Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid alpha-glucosidase. » Sci Transl Med 9(418).

Rio, P., et al. (2017). « Engraftment and in vivo proliferation advantage of gene-corrected mobilized CD34(+) cells from Fanconi anemia patients. » Blood 130(13): 1535-1542.

Romano, O., et al. (2017). « Retroviral Scanning: Mapping MLV Integration Sites to Define Cell-specific Regulatory Regions. » J Vis Exp(123).

Savy, A., et al. (2017). « Impact of Inverted Terminal Repeat Integrity on rAAV8 Production Using the Baculovirus/Sf9 Cells System. » Hum Gene Ther Methods 28(5): 277-289.

Teyssou, E., et al. (2017). « Novel UBQLN2 mutations linked to amyotrophic lateral sclerosis and atypical hereditary spastic paraplegia phenotype through defective HSP70-mediated proteolysis. » Neurobiol Aging 58: 239 e211-239 e220.

Vendomele, J., et al. (2017). « Cellular and Molecular Mechanisms of Anterior Chamber-Associated Immune Deviation (ACAID): What We Have Learned from Knockout Mice. » Front Immunol 8: 1686.

Vermeer, L. S., et al. (2017). « Vectofusin-1, a potent peptidic enhancer of viral gene transfer forms pH-dependent alpha-helical nanofibrils, concentrating viral particles. » Acta Biomater 64: 259-268.

Vidal, P., et al. (2017). « Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression. » Mol Ther.

Vihola, A., et al. (2017). « Diagnostic anoctamin-5 protein defect in patients with ANO5-mutated muscular dystrophy. » Neuropathol Appl Neurobiol.

2016

Aranyi, T., et al. (2016). « Systemic epigenetic response to recombinant lentiviral vectors independent of proviral integration » Epigenetics Chromatin 9: 29.

Armbruster, N., et al. (2016). « Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy. » Mol Ther Methods Clin Dev 3: 16060.

Barbon, E., et al. (2016). « Transposon-mediated Generation of Cellular and Mouse Models of Splicing Mutations to Assess the Efficacy of snRNA-based Therapeutics. » Mol Ther Nucleic Acids 5(11): e392.

Bennett, J., et al. (2016). « Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial » Lancet 388(10045): 661-672

Cavazza, A., A. Miccio, et al. (2016). « Dynamic Transcriptional and Epigenetic Regulation of Human Epidermal Keratinocyte Differentiation. » Stem Cell Reports 6: 618-.

Charton, K., et al. (2016). « Exploiting the CRISPR/Cas9 system to study alternative splicing in vivo: application to titin. » Hum Mol Genet 25(20): 4518-4532.

Cire, S., et al. (2016). « In Vivo Gene Delivery to Lymph Node Stromal Cells Leads to Transgene-specific CD8+ T Cell Anergy in Mice. » Mol Ther 24(11): 1965-1973.

Corre, G., M. Dessainte, et al. (2016). « RCL-Pooling Assay: A Simplified Method for the Detection of Replication-Competent Lentiviruses in Vector Batches Using Sequential Pooling. » Hum Gene Ther 27(2): 202-210.

Cosette, J., et al. (2016). « Bioluminescence-Based Tumor Quantification Method for Monitoring Tumor Progression and Treatment Effects in Mouse Lymphoma Models. » Vis Exp(113).

Costopoulos, M., et al. (2016). « ISOLD: A New Highly Sensitive Interleukin Score for Intraocular Lymphoma Diagnosis. » Ophthalmology 123(7): 1626-1628.

D'Costa, S., V. Blouin, et al. (2016). « Practical utilization of recombinant AAV vector reference standards: focus on vector genomes titration by free ITR qPCR. » Mol Ther Methods Clin Dev 5: 16019.

El Shafey, N., et al. (2016). « Inhibition of the myostatin/Smad signaling pathway by short decorin-derived peptides » Exp Cell Res 341(2): 187-195.

Galy, A. (2016). « Like Angler Fish, CAARs Lure Their Prey » Mol Ther 24(8): 1339-1341.

Gentil, C., et al. (2016). « Dystrophin Threshold Level Necessary for Normalization of Neuronal Nitric Oxide Synthase, Inducible Nitric Oxide Synthase, and Ryanodine Receptor-Calcium Release Channel Type 1 Nitrosylation in Golden Retriever Muscular Dystrophy Dystrophinopathy. » Hum Gene Ther 27(9): 712-726.

Gregoire, S., C. Terrada, et al. (2016). « Treatment of Uveitis by In Situ Administration of Ex Vivo-Activated Polyclonal Regulatory T Cells. » J Immunol 196(5): 2109-2118.

Holic, N. and D. Fenard (2016). « Production of Retrovirus-Based Vectors in Mildly Acidic pH Condition » Methods Mol Biol 1448: 41-48.

Hudry, E., C. Martin, et al. (2016). « Exosome-associated AAV vector as a robust and convenient neuroscience tool. » Gene Ther 23(4): 380-392.

Israeli, D., et al. (2016). « Circulating miRNAs are generic and versatile therapeutic monitoring biomarkers in muscular dystrophies » Sci Rep 6: 28097.

Kutchukian, C., et al. (2016). « Phosphatidylinositol 3-kinase inhibition restores Ca2+ release defects and prolongs survival in myotubularin-deficient mice. » Proc Natl Acad Sci U S A 113(50): 14432-14437.

Lawlor, M. W., et al. (2016). « Skeletal Muscle Pathology in X-Linked Myotubular Myopathy: Review With Cross-Species Comparisons » J Neuropathol Exp Neurol 75(2): 102-110.

Majdoul, S., A. K. Seye, et al. (2016). « Molecular Determinants of Vectofusin-1 and Its Derivatives for the Enhancement of Lentivirally Mediated Gene Transfer into Hematopoietic Stem/Progenitor Cells. » J Biol Chem 291(5): 2161-2169.

Mansour, R., et al. (2016). « Expression of myotubularins in blood platelets: Characterization and potential diagnostic of X-linked myotubular myopathy » Biochem Biophys Res Commun 476(3): 167-173.

Martinet, J., et al. (2016). « Induction of Hematopoietic Microchimerism by Gene-Modified BMT Elicits Antigen-Specific B and T Cell Unresponsiveness toward Gene Therapy Products. » Front Immunol 7: 360.

Masat, E., et al. (2016). « Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients. » Sci Rep 6: 36182.

Merten, O. W. and J. F. Wright (2016). « Towards routine manufacturing of gene therapy drug » Mol Ther Methods Clin Dev 3: 16021.

Merten, O. W., M. Hebben, et al. (2016). « Production of lentiviral vectors. » Mol Ther Methods Clin Dev 3: 16017.

Richard, I., et al. (2016). « Natural history of LGMD2A for delineating outcome measures in clinical trials. » Ann Clin Transl Neurol 3(4): 248-265.

Richard, I., et al. (2016). « 216th ENMC international workshop: Clinical readiness in FKRP related myopathies » January 15-17, 2016 Naarden, The Netherlands. Neuromuscul Disord 26(10): 717-724.

Rivera-Munoz, P., V. Abramowski, et al. (2016). « Lymphopoiesis in transgenic mice over-expressing Artemis. » Gene Ther 23(2): 176-186.

Romano, O., C. Peano, et al. (2016). « Transcriptional, epigenetic and retroviral signatures identify regulatory regions involved in hematopoietic lineage commitment. » Sci Rep 6: 24724.

Ronzitti, G., et al. (2016). « A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome. » Mol Ther Methods Clin Dev 3: 16049.

Sabha, N., et al. (2016). « PIK3C2B inhibition improves function and prolongs survival in myotubular myopathy animal models. » J Clin Invest 126(9): 3613-3625.

Verhoeyen, E., et al. (2016). « Twelfth Annual Meeting of the French Society of Cell and Gene Therapy. » Hum Gene Ther 27(7): 555-558.

Vermeer, L. S., et al. (2016). « Simultaneous Analysis of Secondary Structure and Light Scattering from Circular Dichroism Titrations: Application to Vectofusin-1. » Sci Rep 6: 39450.

2015

Aalbers, C. J., L. Bevaart, et al. (2015). « Preclinical Potency and Biodistribution Studies of an AAV 5 Vector Expressing Human Interferon-beta (ART-I02) for Local Treatment of Patients with Rheumatoid Arthritis. » PLoS One 10(6): e0130612.

Ben Abdelwahed Bagga, R., S. Donnou, et al. (2015). « Mouse models of primary central nervous system lymphomas: tools for basing funding and therapeutic strategies. » J Neurooncol 121(1): 9-18.

Boisgerault, F. and F. Mingozzi (2015). « The Skeletal Muscle Environment and Its Role in Immunity and Tolerance to AAV Vector-Mediated Gene Transfer. » Curr Gene Ther 15(4): 381-394.

Cantore, A., M. Ranzani, et al. (2015). « Liver-directed lentiviral gene therapy in a dog model of hemophilia B. » Sci Transl Med 7(277): 277ra228.

Charton, K., J. Sarparanta, et al. (2015). « CAPN3-mediated processing of C-terminal titin replaced by pathological cleavage in titinopathy. » Hum Mol Genet 24(13): 3718-3731.

Cheever, T. R., D. Berkley, et al. (2015). « Perspectives on best practices for gene therapy programs. » Hum Gene Ther 26(3): 127-133.

Childers, M. K., A. H. Beggs, et al. (2015). « Gene replacement rescues severe muscle pathology and prolongs survival in myotubularin-deficient mice and dogs. » Ann Transl Med 3(17): 257.

Cosette, J., A. Moussy, et al. (2015). « Single Cell Dynamics Causes Pareto-Like Effect in Stimulated T Cell Populations. » Sci Rep 5: 17756.

De Cid, R., R. Ben Yaou, et al. (2015). « A new titinopathy: Childhood-juvenile onset Emery-Dreifuss-like phenotype without cardiomyopathy. » Neurology 85(24): 2126-2135.

Droz-Georget Lathion, S., A. Rochat, et al. (2015). « A single epidermal stem cell strategy for safe ex vivo gene therapy. » EMBO Mol Med.

Ferrand, M., S. Da Rocha, et al. (2015). « Serotype-specific Binding Properties and Nanoparticle Characteristics Contribute to the Immunogenicity of rAAV1 Vectors. » Mol Ther.

Hacein-Bey Abina, S., H. B. Gaspar, et al. (2015). « Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome. » JAMA 313(15): 1550-1563.

Han, S. O., S. Li, et al. (2015). « Enhanced efficacy from gene therapy in Pompe disease using coreceptor blockade. » Hum Gene Ther 26(1): 26-35.

Junge, N., F. Mingozzi, et al. (2015). « Adeno-associated virus vector-based gene therapy for monogenetic metabolic diseases of the liver. » Pediatr Gastroenterol Nutr 60(4): 433-440.

Lecomte, E., B. Tournaire, et al. (2015). « Advanced Characterization of DNA Molecules in rAAV Vector Preparations by Single-stranded Virus Next-generation Sequencing. » Mol Ther Nucleic Acids 4: e260.

Marini, B., A. Kertesz-Farkas, et al. (2015). « Nuclear architecture dictates HIV-1 integration site selection. » Nature.

Meliani, A., C. Leborgne, et al. (2015). « Determination of anti-adeno-associated virus vector neutralizing antibody titer with an in vitro reporter system. » Hum Gene Ther Methods 26(2): 45-53.

Merten, O. W. (2015). « Advances in cell culture: anchorage dependence. » Philos Trans R Soc Lond B Biol Sci 370(1661): 20140040.

Mingozzi, F. and H. Buning (2015). « Adeno-Associated Viral Vectors at the Frontier between Tolerance and Immunity. » Front Immunol 6: 120.

Molina-Estevez, F. J., A. Nowrouzi, et al. (2015). « Lentiviral-Mediated Gene Therapy in Fanconi Anemia-A Mice Reveals Long-Term Engraftment and Continuous Turnover of Corrected HSCs. » Curr Gene Ther 15(6): 550-562.

Mora, M., C. Angelini, et al. (2015). « The EuroBioBank Network: 10 years of hands-on experience of collaborative, transnational biobanking for rare diseases. » Eur J Hum Genet 23(9): 1116-1123.

Nectoux, J., R. de Cid, et al. (2015). « Detection of TRIM32 deletions in LGMD patients analyzed by a combined strategy of CGH array and massively parallel sequencing. » Eur J Hum Genet 23(7): 929-934.

Poletti, V., A. Delli Carri, et al. (2015). « Genome-Wide Definition of Promoter and Enhancer Usage during Neural Induction of Human Embryonic Stem Cells. » PLoS One 10(5): e0126590.

Pryadkina, M., W. Lostal, et al. (2015). « A comparison of AAV strategies distinguishes overlapping vectors for efficient systemic delivery of the 6.2 kb Dysferlin coding sequence. » Mol Ther Methods Clin Dev 2: 15009.

Ribera, A., V. Haurigot, et al. (2015). « Biochemical, histological and functional correction of mucopolysaccharidosis type IIIB by intra-cerebrospinal fluid gene therapy. » Hum Mol Genet 24(7): 2078-2095.

Rouillon, J., J. Poupiot, et al. (2015). « Serum proteomic profiling reveals fragments of MYOM3 as potential biomarkers for monitoring the outcome of therapeutic interventions in muscular dystrophies. » Hum Mol Genet 24(17): 4916-4932.

Saliba, J., C. Saint-Martin, et al. (2015). « Germline duplication of ATG2B and GSKIP predisposes to familial myeloid malignancies. » Nat Genet.

Wary, C., N. Azzabou, et al. (2015). « Quantitative NMRI and NMRS identify augmented disease progression after loss of ambulation in forearms of boys with Duchenne muscular dystrophy. » NMR Biomed 28(9): 1150-1162.

Zocevic, A., J. Rouillon, et al. (2015). « Evaluation of the serum matrix metalloproteinase-9 as a biomarker for monitoring disease progression in Duchenne muscular dystrophy. » Neuromuscul Disord 25(5): 444-446

2014

Allenbach, Y., W. Chaara, et al. (2014). « Th1 response and systemic treg deficiency in inclusion body myositis. » PLoS One 9(3): e88788.

Basner-Tschakarjan, E. and F. Mingozzi (2014). « Cell-Mediated Immunity to AAV Vectors, Evolving Concepts and Potential Solutions. » Front Immunol 5: 350.

Brayet, J., F. Zehraoui, et al. (2014). « Towards a piRNA prediction using multiple kernel fusion and support vector machine. » Bioinformatics 30(17): i364-i370.

Childers, M. K., R. Joubert, et al. (2014). « Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy. » Sci Transl Med 6(220): 220ra210.

Chuah, M. K., I. Petrus, et al. (2014). « Liver-specific transcriptional modules identified by genome-wide in silico analysis enable efficient gene therapy in mice and non-human primates. » Mol Ther 22(9): 1605-1613.

Cire, S., S. Da Rocha, et al. (2014). « Immunization of Mice with Lentiviral Vectors Targeted to MHC Class II+ Cells Is Due to Preferential Transduction of Dendritic Cells In Vivo. » LoS One 9(7): e101644.

Clermont, D., S. Santoni, et al. (2014). « Assessment of DNA encapsulation, a new room-temperature DNA storage method. » Biopreserv Biobank 12(3): 176-183.

Corre, G., D. Stockholm, et al. (2014). « Stochastic fluctuations and distributed control of gene expression impact cellular memory. » PLoS One 9(12): e115574.

Denard, J., C. Jenny, et al. (2014). « Different protein composition and functional properties of adeno-associated virus-6 vector manufactured from the culture medium and cell lysates. » Molecular Therapy — Methods & Clinical Development 1.

Evila, A., A. Vihola, et al. (2014). « Atypical phenotypes in titinopathies explained by second titin mutations. » Ann Neurol 75(2): 230-240.

Ferrand, M., A. Galy, et al. (2014). « A dystrophic muscle broadens the contribution and activation of immune cells reacting to rAAV gene transfer. » Gene Ther.

Fraysse, B., A. Vignaud, et al. (2014). « Acute effect of androgens on maximal force-generating capacity and electrically evoked calcium transient in mouse skeletal muscles. » Steroids.

Goddard, M. A., E. Burlingame, et al. (2014). « Gait characteristics in a canine model of X-linked myotubular myopathy. » J Neurol Sci 346: 221-226.

Holic, N., A. K. Seye, et al. (2014). « Influence of Mildly Acidic pH Conditions on the Production of Lentiviral and Retroviral Vectors. » Hum Gene Ther Clin Dev 25(3): 178-185.

Ingrao, D., S. Majdoul, et al. (2014). « Concurrent measures of fusion and transduction efficiency of primary CD34+ cells with human immunodeficiency virus 1-based lentiviral vectors reveal different effects of transduction enhancers. » Hum Gene Ther Methods 25(1): 48-56.

Jeanson-Leh, L., J. Lameth, et al. (2014). « Serum Profiling Identifies Novel Muscle miRNA and Cardiomyopathy-Related miRNA Biomarkers in Golden Retriever Muscular Dystrophy Dogs and Duchenne Muscular Dystrophy Patients. » Am J Pathol.

Lawlor, M. W., M. G. Viola, et al. (2014). « Differential Muscle Hypertrophy Is Associated with Satellite Cell Numbers and Akt Pathway Activation Following Activin Type IIB Receptor Inhibition in Mtm1 p.R69C Mice. » Am J Pathol 184(6): 1831-1842.

Le Guiner, C., M. Montus, et al. (2014). « Forelimb Treatment in a Large Cohort of Dystrophic Dogs Supports Delivery of a Recombinant AAV for Exon Skipping in Duchenne Patients. » Mol Ther.

Mavilio, F. (2014). « Repairing without cutting: a safer alternative to gene correction? » Mol Ther 22(4): 690-691.

Moiani, A., J. D. Suerth, et al. (2014). « Genome-wide analysis of alpharetroviral integration in human hematopoietic stem/progenitor cells. » Genes (Basel) 5(2): 415-429.

Monjaret, F., N. Bourg, et al. (2014). « Cis-splicing and Translation of the Pre-Trans-splicing Molecule Combine With Efficiency in Spliceosome-mediated RNA Trans-splicing. » Mol Ther.

Mouisel, E., K. Relizani, et al. (2014). « Myostatin is a key mediator between energy metabolism and endurance capacity of skeletal muscle. » Am J Physiol Regul Integr Comp Physiol 307(4): R444-454.

Nathwani, A. C., U. M. Reiss, et al. (2014). « Long-term safety and efficacy of factor IX gene therapy in hemophilia B. » Engl J Med 371(21): 1994-2004.

Neildez-Nguyen, T. M., J. Bigot, et al. (2014). « Hypoxic culture conditions enhance the generation of regulatory T cells. » Immunology.

Relizani, K., E. Mouisel, et al. (2014). « Blockade of ActRIIB signaling triggers muscle fatigability and metabolic myopathy. » Mol Ther.

Rodriguez, E. G., R. Lefebvre, et al. (2014). « Phosphoinositide substrates of myotubularin affect voltage-activated Ca(2)(+) release in skeletal muscle. » Pflugers Arch 466(5): 973-985.

Rouillon, J., A. Zocevic, et al. (2014). « Proteomics profiling of urine reveals specific titin fragments as biomarkers of Duchenne muscular dystrophy. » Neuromuscul Disord.

Sarwal, A., M. S. Cartwright, et al. (2014). « Ultrasound assessment of the diaphragm: Preliminary study of a canine model of X-linked myotubular myopathy. » Muscle Nerve 50(4): 607-609.

Turchiano, G., M. C. Latella, et al. (2014). « Genomic analysis of sleeping beauty transposon integration in human somatic cells. » PLoS One 9(11): e112712.

2013

Aiuti, A., L. Biasco, et al. (2013). « Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome. » Science 341(6148): 1233151.

Andre-Schmutz, I., L. Dal-Cortivo, et al. (2013). « Genotoxic signature in cord blood cells of newborns exposed in utero to a Zidovudine-based antiretroviral combination. » J Infect Dis 208(2): 235-243.

Ben Abdelwahed, R., J. Cosette, et al. (2013). « Lymphoma B-cell responsiveness to CpG-DNA depends on the tumor microenvironment. » J Exp Clin Cancer Res 32: 18.

Ben Abdelwahed, R., S. Donnou, et al. (2013). « Preclinical study of Ublituximab, a Glycoengineered anti-human CD20 antibody, in murine models of primary cerebral and intraocular B-cell lymphomas. » Invest Ophthalmol Vis Sci 54(5): 3657-3665.

Biondi, O., M. Villemeur, et al. (2013). « Dual effects of exercise in dysferlinopathy. » Am J Pathol 182(6): 2298-2309.

Blandin, G., S. Marchand, et al. (2013). « A human skeletal muscle interactome centered on proteins involved in muscular dystrophies: LGMD interactome. » Skelet Muscle 3(1): 3.

Boisgerault, F., D. A. Gross, et al. (2013). « Prolonged gene expression in muscle is achieved without active immune tolerance using MicrorRNA 142.3p-regulated rAAV gene transfer. » Hum Gene Ther 24(4): 393-405.

Cavazza, A., A. Moiani, et al. (2013). « Mechanisms of retroviral integration and mutagenesis. » Hum Gene Ther 24(2): 119-131.

Cavazza, A., F. Cocchiarella, et al. (2013). « Self-inactivating MLV vectors have a reduced genotoxic profile in human epidermal keratinocytes. » Gene Ther.

Charrier, S., M. Blundell, et al. (2013). « Wiskott-Aldrich syndrome protein-deficient hematopoietic cells can be efficiently mobilized by granulocyte colony-stimulating factor. » Haematologica 98(8): 1300-1308.

Coluccio, A., F. Miselli, et al. (2013). « Targeted Gene Addition in Human Epithelial Stem Cells by Zinc-finger Nuclease-mediated Homologous Recombination. » Mol Ther 21(9): 1695-1704.

Decostre, V., A. Vignaud, et al. (2013). « Longitudinal in vivo muscle function analysis of the DMSXL mouse model of myotonic dystrophy type 1. » Neuromuscul Disord.

Denard, J., B. Marolleau, et al. (2013). « C-reactive protein (CRP) is essential for efficient rAAV-1 and rAAV-6 systemic transduction in mice. » Virol.

Di Nunzio, F., T. Fricke, et al. (2013). « Nup153 and Nup98 bind the HIV-1 core and contribute to the early steps of HIV-1 replication. » Virology 440(1): 8-18.

Fayssoil, A., G. Renault, et al. (2013). « Cardiac characterization of mdx mice using high-resolution doppler echocardiography. » J Ultrasound Med 32(5): 757-761.

Fayssoil, A., G. Renault, et al. (2013). « Cardiac Characterization of sgca-Null Mice Using High Resolution Echocardiography. » Neurol Int 5(4): e22.

Fenard, D., D. Ingrao, et al. (2013). « Vectofusin-1, a new viral entry enhancer, strongly promotes lentiviral transduction of human hematopoietic stem cells. » Mol Ther Nucleic Acids 2: e90.

Fenard, D., S. Genries, et al. (2013). « Infectivity enhancement of different HIV-1-based lentiviral pseudotypes in presence of the cationic amphipathic peptide LAH4-L1. » J Virol Methods 189(2): 375-378.

Fisson, S., H. Ouakrim, et al. (2013). « Cytokine profile in human eyes: contribution of a new cytokine combination for differential diagnosis between intraocular lymphoma or uveitis. » PLoS One 8(2): e52385.

Hirsch, M. L., C. Li, et al. (2013). « Oversized AAV Transduction is Mediated via a DNA-PKcs Independent, Rad51C-dependent Repair Pathway. » Mol Ther.

Hui, D. J., E. Basner-Tschakarjan, et al. (2013). « Modulation of CD8 T cell responses to AAV vectors with IgG-derived MHC class II epitopes. » Mol Ther.

Joubert, R., A. Vignaud, et al. (2013). « Site-specific Mtm1 mutagenesis by an AAV-Cre vector reveals that myotubularin is essential in adult muscle. » Hum Mol Genet 22(9): 1856-1866.

Kaufmann, K. B., H. Buning, et al. (2013). « Gene therapy on the move. » EMBO Mol Med.

Lawlor, M. W., D. Armstrong, et al. (2013). « Enzyme replacement therapy rescues weakness and improves muscle pathology in mice with X-linked myotubular myopathy. » Hum Mol Genet 22(8): 1525-1538.

Marek, M., C. Romier, et al. (2013). « Baculovirus VP1054 Is an Acquired Cellular PURalpha, a Nucleic Acid-Binding Protein Specific for GGN Repeats. » J Virol 87(15): 8465-8480.

Masat, E., G. Pavani, et al. (2013). « Humoral immunity to AAV vectors in gene therapy: challenges and potential solutions. » Discov Med 15(85): 379-389.

Mingozzi, F., X. M. Anguela, et al. (2013). « Overcoming Preexisting Humoral Immunity to AAV Using Capsid Decoys. » Sci Transl Med 5(194): 194ra192.

Moiani, A., A. Miccio, et al. (2013). « Deletion of the LTR enhancer/promoter has no impact on the integration profile of MLV vectors in human hematopoietic progenitors. » PLoS One 8(1): e55721.

Monjaret, F., L. Suel-Petat, et al. (2013). « The phenotype of dysferlin-deficient mice is not rescued by adeno-associated virus-mediated transfer of anoctamin 5. » Hum Gene Ther Clin Dev 24(2): 65-76.

Nahdi, I., R. B. Abdelwahed, et al. (2013). « Herpesvirus detection and cytokine levels (IL-10, IL-6, and IFN-gamma) in ocular fluid from tunisian immunocompetent patients with uveitis. » J Med Virol.

Paldi, A. (2013). « Effects of the in vitro manipulation of stem cells: epigenetic mechanisms as mediators of induced metabolic fluctuations. » Epigenomics 5(4): 429-437.

Roudaut, C., F. Le Roy, et al. (2013). « Restriction of Calpain3 Expression to the Skeletal Muscle Prevents Cardiac Toxicity and Corrects Pathology in a Murine Model of Limb-Girdle Muscular Dystrophy. » Circulation.

Saliba, J., S. Hamidi, et al. (2013). « Heterozygous and homozygous JAK2(V617F) states modeled by induced pluripotent stem cells from myeloproliferative neoplasm patients. » PLoS One 8(9): e74257.

Silva, A. K., C. Richard, et al. (2013). « Xyloglucan-derivatized films for the culture of adherent cells and their thermocontrolled detachment: a promising alternative to cells sensitive to protease treatment. » Biomacromolecules 14(2): 512-519.

Tremblay, J. P., X. Xiao, et al. (2013). « Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases. » Mol Ther 21(2): 266-268.

van Zwieten, R. W., S. Puttini, et al. (2013). « Assessing dystrophies and other muscle diseases at the nanometer scale by atomic force microscopy. » Nanomedicine (Lond).

Vignier, N., F. Amor, et al. (2013). « Distinctive serum miRNA profile in mouse models of striated muscular pathologies. » PLoS One 8(2): e55281.

Zerbato, M., N. Holic, et al. (2013). « The brown algae Pl.LSU/2 group II intron-encoded protein has functional reverse transcriptase and maturase activities. » LoS One 8(3): e58263.

2012

Carrondo, M. J., P. M. Alves, et al. (2012). « How can measurement, monitoring, modeling and control advance cell culture in industrial biotechnology? » Biotechnol J 7(12): 1522-1529.

Denard, J., C. Beley, et al. (2012). « Human galectin 3 binding protein interacts with recombinant adeno-associated virus type 6. » Virol 86(12): 6620-6631.

Donnou, S., C. Galand, et al. (2012). « Murine models of B-cell lymphomas: promising tools for designing cancer therapies. » Adv Hematol 2012: 701704.

Dowling, J. J., R. Joubert, et al. (2012). « Myotubular myopathy and the neuromuscular junction: a novel therapeutic approach from mouse models. » Dis Model Mech 5(6): 852-859.

Galand, C., S. Donnou, et al. (2012). « Influence of Tumor Location on the Composition of Immune Infiltrate and Its Impact on Patient Survival. Lessons from DCBCL and Animal Models. » Front Immunol 3: 98.

Guiraud, S., L. van Wittenberghe, et al. (2012). « Identification of decorin derived peptides with a zinc dependent anti-myostatin activity. » Neuromuscul Disord 22(12): 1057-1068.

Herson, S., F. Hentati, et al. (2012). « A phase I trial of adeno-associated virus serotype 1-gamma-sarcoglycan gene therapy for limb girdle muscular dystrophy type 2C. » Brain 135(Pt 2): 483-492.

Lafouresse, F., V. Cotta-de-Almeida, et al. (2012). « Wiskott-Aldrich syndrome protein controls antigen-presenting cell-driven CD4+ T-cell motility by regulating adhesion to intercellular adhesion molecule-1. » Immunology 137(2): 183-196.

Lawlor, M. W., M. S. Alexander, et al. (2012). « Myotubularin-deficient myoblasts display increased apoptosis, delayed proliferation, and poor cell engraftment. » Am J Pathol 181(3): 961-968.

Lostal, W., M. Bartoli, et al. (2012). « Lack of correlation between outcomes of membrane repair assay and correction of dystrophic changes in experimental therapeutic strategy in dysferlinopathy. » PLoS One 7(5): e38036.

Marek, M., O. W. Merten, et al. (2012). « Essential C-terminal region of the baculovirus minor capsid protein VP80 binds DNA. » Virol 86(3): 1728-1738.

Mavilio, F. (2012). « Gene therapies need new development models. » Nature 490(7418): 7.

Paldi, A. (2012). « What makes the cell differentiate? » Prog Biophys Mol Biol 110(1): 41-43.

Pathare, A. V., S. Al Zadjali, et al. (2012). « Warfarin pharmacogenetics: polymorphisms of the CYP2C9, CYP4F2, and VKORC1 loci in a genetically admixed Omani population. » Hum Biol 84(1): 67-77.

Pathare, A., M. Al Khabori, et al. (2012). « Warfarin pharmacogenetics: development of a dosing algorithm for Omani patients. » Hum Genet 57(10): 665-669.

Pierson, C. R., A. N. Dulin-Smith, et al. (2012). « Modeling the human MTM1 p.R69C mutation in murine Mtm1 results in exon 4 skipping and a less severe myotubular myopathy phenotype. » Hum Mol Genet 21(4): 811-825.

Soheili, T., E. Gicquel, et al. (2012). « Rescue of sarcoglycan mutations by inhibition of endoplasmic reticulum quality control is associated with minimal structural modifications. » Hum Mutat 33(2): 429-439.

Sudres, M., S. Cire, et al. (2012). « MyD88 signaling in B cells regulates the production of Th1-dependent antibodies to AAV. » Mol Ther 20(8): 1571-1581.

Veron, P., C. Leborgne, et al. (2012). « Humoral and cellular capsid-specific immune responses to adeno-associated virus type 1 in randomized healthy donors. » Immunol 188(12): 6418-6424.

Yamagata, Y., P. Szabo, et al. (2012). « Rapid turnover of DNA methylation in human cells. » Epigenetics 7(2): 141-145.

Yamagata, Y., V. Parietti, et al. (2012). « Lentiviral transduction of CD34(+) cells induces genome-wide epigenetic modifications. » PLoS One 7(11): e48943.

2011

Barde, I., E. Laurenti, et al. (2011). « Lineage- and stage-restricted lentiviral vectors for the gene therapy of chronic granulomatous disease. » Gene Ther 18(11): 1087-1097.

Bechinger, B., V. Vidovic, et al. (2011). « A new family of peptide-nucleic acid nanostructures with potent transfection activities. » J Pept Sci 17(2): 88-93.

Brun-Graeppi, A. K., C. Richard, et al. (2011). « Cell microcarriers and microcapsules of stimuli-responsive polymers. » J Control Release 149(3): 209-224.

Calvez, R., F. Lafouresse, et al. (2011). « The Wiskott-Aldrich syndrome protein permits assembly of a focused immunological synapse enabling sustained T-cell receptor signaling. » Haematologica 96(10): 1415-1423.

Carre-Pierrat, M., A. Lafoux, et al. (2011). « Pre-clinical study of 21 approved drugs in the mdx mouse. » Neuromuscul Disord 21(5): 313-327.

Carrondo, M., A. Panet, et al. (2011). « Integrated strategy for the production of therapeutic retroviral vectors. » Hum Gene Ther 22(3): 370-379.

Charrier, S., M. Ferrand, et al. (2011). « Quantification of lentiviral vector copy numbers in individual hematopoietic colony-forming cells shows vector dose-dependent effects on the frequency and level of transduction. » Gene Ther 18(5): 479-487.

Galibert, L. and O. W. Merten (2011). « Latest developments in the large-scale production of adeno-associated virus vectors in insect cells toward the treatment of neuromuscular diseases. » J Invertebr Pathol 107 Suppl: S80-93.

Galy, A. and A. J. Thrasher (2011). « Gene therapy for the Wiskott-Aldrich syndrome. » Curr Opin Allergy Clin Immunol 11(6): 545-550.

Guiraud, S., D. Alimi-Guez, et al. (2011). « The reverse block copolymer Pluronic 25R2 promotes DNA transfection of skeletal muscle. » Macromol Biosci 11(5): 590-594.

Jeanson-Leh, L., S. Charrier, et al. (2011). « The c.273+11dup genetic change in the WAS gene is a functionally neutral polymorphism. » Eur J Haematol 87(4): 366-371.

Kamen, A. A., M. G. Aucoin, et al. (2011). « An initiative to manufacture and characterize baculovirus reference material. » J Invertebr Pathol 107 Suppl: S113-117.

Lawlor, M. W., B. P. Read, et al. (2011). « Inhibition of activin receptor type IIB increases strength and lifespan in myotubularin-deficient mice. » Am J Pathol 178(2): 784-793.

Marek, M., O. W. Merten, et al. (2011). « Baculovirus VP80 protein and the F-actin cytoskeleton interact and connect the viral replication factory with the nuclear periphery. » J Virol 85(11): 5350-5362.

Merten, O. W., S. Charrier, et al. (2011). « Large-scale manufacture and characterization of a lentiviral vector produced for clinical ex vivo gene therapy application. » Hum Gene Ther 22(3): 343-356.

Mezzina, M. and O. W. Merten (2011). « Adeno-associated viruses. » Methods Mol Biol 737: 211-234.

Monteilhet, V., S. Saheb, et al. (2011). « A 10 patient case report on the impact of plasmapheresis upon neutralizing factors against adeno-associated virus (AAV) types 1, 2, 6, and 8. » Mol Ther 19(11): 2084-2091.

Ni, W., C. Le Guiner, et al. (2011). « Longevity of rAAV vector and plasmid DNA in blood after intramuscular injection in nonhuman primates: implications for gene doping. » Gene Ther 18(7): 709-718.

Poupiot, J., J. Ausseil, et al. (2011). « Methods for noninvasive monitoring of muscle fiber survival with an AAV vector encoding the mSEAP reporter gene. » Methods Mol Biol 709: 63-74.

Sambasivan, R., R. Yao, et al. (2011). « Pax7-expressing satellite cells are indispensable for adult skeletal muscle regeneration. » Development 138(17): 3647-3656.

Stacey, G. N. and O. W. Merten (2011). « Host cells and cell banking. » Methods Mol Biol 737: 45-88.

Tolar, J., J. E. Adair, et al. (2011). « Stem cell gene therapy for fanconi anemia: report from the 1st international Fanconi anemia gene therapy working group meeting. » Mol Ther 19(7): 1193-1198.

2010

Beggs, A.H., J. Bohm, E. Snead, M. Kozlowski, M. Maurer, K. Minor, M.K. Childers, S.M. Taylor, C. Hitte, J.R. Mickelson, L.T. Guo, A.P. Mizisin, A. Buj-Bello, L. Tiret, J. Laporte, and G.D. Shelton, « MTM1 mutation associated with X-linked myotubular myopathy in Labrador Retrievers » Proc Natl Acad Sci U S A, 2010. 107(33): p. 14697-14702

Benkhelifa-Ziyyat, S., S. Bucher, M.A. Zanta-Boussif, J. Pasquet, and O. Danos, « Changes in the accessibility of the HIV-1 Integrase C-terminus in the presence of cellular proteins » Retrovirology, 2010. 7(1): p. 27

Bolduc, V., G. Marlow, K.M. Boycott, K. Saleki, H. Inoue, J. Kroon, M. Itakura, Y. Robitaille, L. Parent, F. Baas, K. Mizuta, N. Kamata, I. Richard, W.H. Linssen, I. Mahjneh, M. de Visser, R. Bashir, and B. Brais, « Recessive Mutations in the Putative Calcium-Activated Chloride Channel Anoctamin 5 Cause Proximal LGMD2L and Distal MMD3 Muscular Dystrophies » Am J Hum Genet, 2010. 86(2): p. 213-221

Boutin, S., V. Monteilhet, P. Veron, C. Leborgne, O. Benveniste, M.F. Montus, and C. Masurier, « Prevalence of serum IgG and neutralizing factors against adeno-associated virus types 1, 2, 5, 6, 8 and 9 in the healthy population: implications for gene therapy using AAV vectors » Hum Gene Ther, 2010. 21(6): p. 704-712

Capron, C., C. Lacout, Y. Lecluse, V. Jalbert, H. Chagraoui, S. Charrier, A. Galy, A. Bennaceur-Griscelli, E. Cramer-Borde, and W. Vainchenker, « A major role of TGF-{beta}1 in the homing capacities of murine hematopoietic stem cell/ progenitors » Blood, 2010. 116(8): p. 1244-1253

Charton, K., N. Daniele, A. Vihola, C. Roudaut, E. Gicquel, F. Monjaret, A. Tarrade, J. Sarparanta, B. Udd, and I. Richard, « Removal of the calpain 3 protease reverses the myopathology in a mouse model for titinopathies » Hum Mol Genet, 2010. 19(23): p. 4608-4624

Claeys, K.G., M. Sozanska, J.J. Martin, E. Lacene, L. Vignaud, D. Stockholm, P. Laforet, B. Eymard, A. Kichler, D. Scherman, T. Voit, and D. Israeli, « DNAJB2 Expression in Normal and Diseased Human and Mouse Skeletal Muscle » Am J Pathol, 2010. 176(6): p. 2901-2910

Hauser, H., M. Carrondo, D. Wirth, A.S. Coroadinha, P. Cruz, H. Falk, R. Schucht, F. Dupont, C. Geny-Fiamma, and O.W. Merten, « Integrated strategy for the production of therapeutic retroviral vectors » Hum Gene Ther, 2010. <em>Online Ahead of Editing: November 2</em>

Klein, E., C. Leborgne, M. Ciobanu, J. Klein, B. Frisch, F. Pons, G. Zuber, D. Scherman, A. Kichler, and L. Lebeau, « Nucleic acid transfer with hemifluorinated polycationic lipids » Biomaterials, 2010. 31(17): p. 4781-8

Klein, E., M. Ciobanu, J. Klein, V. Machi, C. Leborgne, T. Vandamme, B. Frisch, F. Pons, A. Kichler, G. Zuber, and L. Lebeau, « HFP Fluorinated Cationic Lipids for Enhanced Lipoplex Stability and Gene Delivery » Bioconjug Chem, 2010. 21(2): p. 360-71

Krahn, M., N. Wein, M. Bartoli, W. Lostal, S. Courrier, N. Bourg-Alibert, K. Nguyen, C. Vial, N. Streichenberger, V. Labelle, D. DePetris, C. Pécheux, F. Leturcq, P. Cau, I. Richard, and N. Lévy, « A Naturally Occurring Human Minidysferlin Protein Repairs Sarcolemmal Lesions in a Mouse Model of Dysferlinopathy » Science Translational Medicine, 2010. 2(50): p. 50ra69

Lan, Y., B. Langlet-Bertin, V. Abbate, L.S. Vermeer, X. Kong, K.E. Sullivan, C. Leborgne, D. Scherman, R.C. Hider, A.F. Drake, S.S. Bansal, A. Kichler, and A.J. Mason, « Incorporation of 2,3-Diaminopropionic Acid into Linear Cationic Amphipathic Peptides Produces pH-Sensitive Vectors » Chembiochem, 2010. 11(9): p. 1266 – 1272

Langlet-Bertin, B., C. Leborgne, D. Scherman, B. Bechinger, A.J. Mason, and A. Kichler, « Design and Evaluation of Histidine-Rich Amphipathic Peptides for siRNA Delivery » Pharm Res, 2010. 27(7): p. 1426-1436

Laure, L., N. Daniele, L. Suel, S. Marchand, S. Aubert, N. Bourg, C. Roudaut, S. Duguez, M. Bartoli, and I. Richard, « A new pathway encompassing calpain 3 and its newly identified substrate cardiac ankyrin repeat protein is involved in the regulation of the nuclear factor-kappaB pathway in skeletal muscle » FEBS J, 2010. 277(20): p. 4322-4337

Lidgi-Guigui, N., C. Guis, B. Brissault, A. Kichler, C. Leborgne, D. Scherman, S. Labdi, and P.A. Curmi, « Investigation of DNA Condensing Properties of Amphiphilic Triblock Cationic Polymers by Atomic Force Microscopy » Langmuir, 2010. 26(22): p. 17552-17557

Lock, M., S. McGorray, A. Auricchio, E. Ayuso, E.J. Beecham, V. Blouin-Tavel, F. Bosch, M. Bose, B.J. Byrne, T. Caton, J.A. Chiorini, A. Chtarto, K.R. Clark, T. Conlon, C. Darmon, M. Doria, A. Douar, T.R. Flotte, J.D. Francis, A. Francois, M. Giacca, M.T. Korn, I. Korytov, X. Leon, B. Leuchs, G. Lux, C. Melas, H. Mizukami, P. Moullier, M. Müller, K. Ozawa, T. Philipsberg, K. Poulard, C. Raupp, C. Rivière, S.D. Roosendaal, R.J. Samulski, S.M. Soltys, R. Surosky, L. Tenenbaum, D.L. Thomas, B. van Montfort, G. Veres, J.F. Wright, Y. Xu, O. Zelenaia, L. Zentilin, and R.O. Snyder, « Characterization of a Recombinant Adeno-Associated Virus Type 2 Reference Standard Material » Human Gene Therapy, 2010. 21(10): p. 1273-1285

Lostal, W., M. Bartoli, N. Bourg, C. Roudaut, A. Bentaib, K. Miyake, N. Guerchet, F. Fougerousse, P. McNeil, and I. Richard, « Efficient recovery of dysferlin deficiency by dual adeno-associated vectors mediated gene transfer » Hum. Mol. Genet., 2010. 19(10): p. 1897-1907

Moulay, G., C. Masurier, P. Bigey, D. Scherman, and A. Kichler, « Soluble TNF-[alpha] receptor secretion from healthy or dystrophic mice after AAV6-mediated muscle gene transfer » Gene Ther, 2010. 17(11): p. 1400–1410

Sarparanta, J., G. Blandin, K. Charton, A. Vihola, S. Marchand, A. Milic, P. Hackman, E. Ehler, I. Richard, and B. Udd, « Interactions with M-band titin and calpain 3 link myospryn (CMYA5) to tibial and limb-girdle muscular dystrophies » J Biol Chem, 2010. 285(39): p. 30304-30315

Schweizer, M. and O.W. Merten, « Large-Scale Production Means for the Manufacturing of Lentiviral Vectors » Curr Gene Ther, 2010. 10(6): p. 474-486

Wasungu, L., A.L. Marty, M.F. Bureau, A. Kichler, M. Bessodes, J. Teissie, D. Scherman, M.P. Rols, and N. Mignet, « Pre-treatment of cells with pluronic L64 increases DNA transfection mediated by electrotransfer » J Control Release, 2010. 149(2): p. 117-125.]]></content:encoded>

2009

Andriola Silva, A.K., Richard, C., Bessodes, M., Scherman, D. and Merten, O.-W.: « Growth Factor Delivery Approaches in Hydrogels » Biomacromolecules 10 (2009) 9-18.

Bouazza, B., Kratassiouk, G., Gjata, B., Perie, S., Guily, J.L., Butler-Browne, G.S. andSvinartchouk, F.: « Analysis of growth factor expression in affected and unaffected muscles of oculo-pharyngeal muscular dystrophy (OPMD) patients: A pilot study » Neuromuscul Disord 19 (2009) 199-206.

Ciron, C., Cressant, A., Roux, F., Raoul, S., Cherel, Y., Hantraye, P., Deglon, N., Schwartz, B.,Barkats, M., Heard, J.M., Tardieu, M., Moullier, P. and Colle, M.A.: « Human alpha-Iduronidase Gene Transfer Mediated by Adeno-Associated Virus Types 1, 2, and 5 in the Brain of Nonhuman Primates: Vector Diffusion and Biodistribution » Hum Gene Ther 20 (2009) 350-360.

Denard, J., Rundwasser, S., Laroudie, N., Gonnet, F., Naldini, L., Radrizzani, M., Galy, A., Merten, O.-W., Danos, O. and Svinartchouk, F.: « Quantitative proteomic analysis of lentiviral vectors using two dimensional gel electrophoresis » Proteomics 9 (2009) 3666-3676.

Duque, S., Joussemet, B., Riviere, C., Marais, T., Dubreil, L., Douar, A.M., Fyfe, J., Moullier, P., Colle, M.A. and Barkats, M.: « Intravenous Administration of Self-complementary AAV9 Enables Transgene Delivery to Adult Motor Neurons » Mol Ther 17 (2009) 1187–1196.

Gérard, A., Ghiotto, M., Fos, C., Guittard, G., Compagno, D., Galy, A., Lemay, S., Olive, D. and Nunès, J.A.: « Dok-4 is a novel negative regulator of T cell activation » J Immunol 182 (2009) 7681-7689.

Gerard, X., Vignaud, L., Charkes, S., Pinset, C., Scherman, D., Kichler, A. and Israeli, D.: « Real-time monitoring of cell transplantation in mouse dystrophic muscles by a secreted alkaline phosphatase reporter gene » Gene Ther 16 (2009) 815–819.

Goizet, C., Boukhris, A., Durr, A., Beetz, C., Truchetto, J., Tesson, C., Tsaousidou, M., Forlani, S., Guyant-Marechal, L., Fontaine, B., Guimaraes, J., Isidor, B., Chazouilleres, O., Wendum, D.,Grid, D., Chevy, F., Chinnery, P.F., Coutinho, P., Azulay, J.P., Feki, I., Mochel, F., Wolf, C., Mhiri, C., Crosby, A., Brice, A. and Stevanin, G.: « CYP7B1 mutations in pure and complex forms of hereditary spastic paraplegia type 5 » Brain 132 (2009) 1589-600.

Jedidi, A., Marty, C., Oligo, C., Jeanson-Leh, L., Ribeil, J.A., Casadevall, N., Galy, A., Vainchenker, W. and Villeval, J.L.: « Selective reduction of JAK2V617F-dependent cell growth by siRNA / shRNA and its reversal by cytokines » Blood 114 (2009) 1842-1851.

Laure, L., Suel, L., Roudaut, C., Bourg, N., Ouali, A., Bartoli, M., Richard, I. and Daniele, N.: « Cardiac ankyrin repeat protein is a marker of skeletal muscle pathological remodelling » Febs J 276 (2009) 669-84.

Le Roy, F., Charton, K., Lorson, C.L. and Richard, I.: « RNA-targeting approaches for neuromuscular diseases » Trends Mol Med Available online 10 November (2009).

Leclerc, X., Danos, O., Scherman, D. and Kichler, A.: « A comparison of synthetic oligodeoxynucleotides, DNA fragments and AAV-1 for targeted episomal and chromosomal gene repair » BMC Biotechnol 9 (2009) 35.

Mantovani, J., Charrier, S., Eckenberg, R., Saurin, W., Danos, O., Perea, J. and Galy, A.: « Diverse genomic integration of a lentiviral vector developed for the treatment of Wiskott-Aldrich syndrome » J Gene Med 11 (2009) 645 – 654.

Marangoni, F., Bosticardo, M., Charrier, S., Draghici, E., Locci, M., Scaramuzza, S., Panaroni, C., Ponzoni, M., Sanvito, F., Doglioni, C., Liabeuf, M., Gjata, B., Montus, M., Siminovitch, K., Aiuti, A., Naldini, L., Dupre, L., Roncarolo, M.G., Galy, A. and Villa, A.: « Evidence for Long-term Efficacy and Safety of Gene Therapy for Wiskott-Aldrich Syndrome in Preclinical Models » Mol Ther 17 (2009) 1073–1082.

Megarbane, H., Florence, J., Oliver Sass, J., Schwonbeck, S., Foglio, M., de Cid, R., Cure, S.,Saker, S., Megarbane, A. and Fischer, J.: « An Autosomal-Recessive Form of Cutis Laxa Is Due to Homozygous Elastin Mutations, and the Phenotype May Be Modified by a Heterozygous Fibulin 5 Polymorphism » J Invest Dermatol 129 (2009) 1650–1655.

Mellgren, R.L., Miyake, K., Kramerova, I., Spencer, M.J., Bourg, N., Bartoli, M., Richard, I., Greer, P.A. and McNeil, P.L.: « Calcium-dependent plasma membrane repair requires m- or mu-calpain, but not calpain-3, the proteasome, or caspases » Biochim Biophys Acta Available online 23 September (2009).

Modlich, U., Navarro, S., Zychlinski, D., Maetzig, T., Knoess, S., Brugman, M.H., Schambach, A.,Charrier, S., Galy, A., Thrasher, A., Bueren, J. and Baum, C.: « Insertional transformation of hematopoietic cells by selfinactivating lentiviral and gammaretroviral vectors » Mol Ther (Published online 11 August 2009).

Oudot, T., Lesueur, F., Guedj, M., de Cid, R., McGinn, S., Heath, S., Foglio, M., Prum, B., Lathrop, M., Prud'homme, J.F. and Fischer, J.: « An Association Study of 22 Candidate Genes in Psoriasis Families Reveals Shared Genetic Factors with Other Autoimmune and Skin Disorders » J Invest Dermatol Published online 25 June (2009).

Tazir, M., Ali-Pacha, L., M'Zahem, A., Delaunoy, J.P., Fritsch, M., Nouioua, S., Benhassine, T., Assami, S., Grid, D., Vallat, J.M., Hamri, A. and Koenig, M.: « Ataxia with oculomotor apraxia type 2: A clinical and genetic study of 19 patients » J Neurol Sci 278 (2009) 77-81.

Tazir, M., Nouioua, S., Magy, L., Huehne, K., Assami, S., Urtizberea, A., Grid, D., Hamadouche, T., Rautenstrauss, B. and Vallat, J.-M.: « Phenotypic variability in giant axonal neuropathy » Neuromuscular Disorders 19 (2009) 270-274.

Verburg, E., Murphy, R.M., Richard, I. and Lamb, G.D.: « Involvement of calpains in Ca2+-induced disruption of excitation-contraction coupling in mammalian skeletal muscle fibers » Am J Physiol Cell Physiol 296 (2009) C1115-C1122.

Veron, P., Boutin, S., Martin, S., Chaperot, L., Plumas, J., Davoust, J. and Masurier, C.: « Highly efficient transduction of human plasmacytoid dendritic cells without phenotypic and functional maturation » J Transl Med 7 (2009) 10.

Zanta-Boussif, M.A., Charrier, S., Brice-Ouzet, A., Martin, S., Opolon, P., Thrasher, A.J., Hope, T.J. and Galy, A.: « Validation of a mutated PRE sequence allowing high and sustained transgene expression while abrogating WHV-X protein synthesis: application to the gene therapy of WAS » Gene Ther 16 (2009) 605–619.

2008

Agbulut, O., Vignaud, A., Hourde, C., Mouisel, E., Fougerousse, F., Butler-Browne, G. and Ferry, A.: « Slow myosin heavy chain expression in the absence of muscle activity » Am J Physiol Cell Physiol Published Online October 22 2008.

Amaral, A.I., Coroadinha, A.S., Merten, O.W. and Alves, P.M.: « Improving retroviral vectors production: Role of carbon sources in lipid biosynthesis » J Biotechnol 138 (2008) 57-66.

Bacquet, C., Imamura, T., Gonzalez, C.A., Conejeros, I., Kausel, G., Neildez-Nguyen, T.M.,Paldi, A. and Gallardo, M.H.: « Epigenetic processes in a tetraploid mammal » Mamm Genome 19 (2008) 439-447.

Bartoli, M., Gicquel, E., Barrault, L., Soheili, T., Malissen, M., Malissen, B., Vincent-lacaze, N., Perez, N., Udd, B., Danos, O. and Richard, I.: « Mannosidase I inhibition rescues the human {alpha}-sarcoglycan R77C recurrent mutation » Hum. Mol. Genet. 17 (2008) 1214-1221.

Benayoun, B., Baghdiguian, S., Lajmanovich, A., Bartoli, M., Daniele, N., Gicquel, E., Bourg, N., Raynaud, F., Pasquier, M.A., Suel, L., Lochmuller, H., Lefranc, G. and Richard, I.: « NF-{kappa}B-dependent expression of the antiapoptotic factor c-FLIP is regulated by calpain 3, the protein involved in limb-girdle muscular dystrophy type 2A » Faseb J 22 (2008) 1521-1529.

Benjelloun, F., Garrigue, A., Demerens-de Chappedelaine, C., Soulas-Sprauel, P., Malassis-Seris, M., Stockholm, D., Hauer, J., Blondeau, J., Riviere, J., Lim, A., Le Lorc'h, M., Romana, S., Brousse, N., Paques, F., Galy, A., Charneau, P., Fischer, A., de Villartay, J.P. and Cavazzana-Calvo, M.: « Stable and Functional Lymphoid Reconstitution in Artemis-deficient Mice Following Lentiviral Artemis Gene Transfer Into Hematopoietic Stem Cells » Mol Ther 16 (2008) 490–1499.

Boguslaw, A.K., Kirillova, I., Richard, R.E., Israeli, D. and Yablonka-Reuveni, Z.: « FGFR4 and its novel splice form in myogenic cells: interplay of glycosylation and tyrosine phosphorylation » J Cell Physiol 215 (2008) 803 – 817.

Buj-Bello, A., Fougerousse, F., Schwab, Y., Messaddeq, N., Spehner, D., Pierson, C.R., Durand, M., Kretz, C., Danos, O., Douar, A.M., Beggs, A.H., Schultz, P., Montus, M., Denefle, P. and Mandel, J.L.: « AAV-mediated intramuscular delivery of myotubularin corrects the myotubular myopathy phenotype in targeted murine muscle and suggests a function in plasma membrane homeostasis » Hum Mol Genet 17 (2008) 2132-2143.

Claus, S., Fischer, J., Megarbane, H., Megarbane, A., Jobard, F., Debret, R., Peyrol, S., Saker, S., Devillers, M., Sommer, P. and Damour, O.: « A p.C217R Mutation in Fibulin-5 from Cutis Laxa Patients Is Associated with Incomplete Extracellular Matrix Formation in a Skin Equivalent Model » J Invest Dermatol 128 (2008) 1442–1450.

Coulis, G., Becila, S., Herrera-Mendez, C.H., Sentandreu, M.A., Raynaud, F., Richard, I., Benyamin, Y. and Ouali, A.: « Calpain 1 binding capacities of the N1-line region of titin are significantly enhanced by physiological concentrations of calcium » Biochemistry 47 (2008) 9174-83.

Delaguillaumie, A., Marin-Esteban, V., Setterblad, N., Jeanson-Leh, L., Assier, E., Gelin, C., Charron, D., Galy, A. and Mooney, N.: « Contrasting cytoskeletal regulation of MHC class II peptide presentation by human B cells or dendritic cells » Eur J Immunol 38 (2008) 1096-1105.

Delluc-Clavieres, A., Le Bec, C., Van den Berghe, L., Conte, C., Allo, V., Danos, O. and Prats, A.C.: « Efficient gene transfer in skeletal muscle with AAV-derived bicistronic vector using the FGF-1 IRES » Gene Ther 15 (2008) 1090–1098.

Galy, A., Roncarolo, M.G. and Thrasher, A.J.: « Development of lentiviral gene therapy for Wiskott Aldrich syndrome » Expert Opin Biol Ther 8 (2008) 181-190.

Gonnet, F., Bouazza, B., Millot, G.A., Ziaei, S., Garcia, L., Butler-Browne, G.S., Mouly, V., Tortajada, J., Danos, O. and Svinartchouk, F.: « Proteome analysis of differentiating human myoblasts by dialysis-assisted two-dimensional gel electrophoresis (DAGE) » Proteomics 8 (2008) 264-278.

Hackman, P., Marchand, S., Sarparanta, J., Vihola, A., Penisson-Besnier, I., Eymard, B., Pardal-Fernandez, J.M., Hammouda, E.H., Richard, I., Illa, I. and Udd, B.: « Truncating mutations in C-terminal titin may cause more severe tibial muscular dystrophy (TMD) » Neuromuscul Disord Published online 22 October 2008.

Hamadouche, T., Poitelon, Y., Genin, E., Chaouch, M., Tazir, M., Kassouri, N., Nouioua, S., Chaouch, A., Boccaccio, I., Benhassine, T., De Sandre-Giovannoli, A., Grid, D., Levy, N. and Delague, V.: « Founder Effect and Estimation of the Age of the c.892C>T (p.Arg298Cys) Mutation in LMNA Associated to Charcot-Marie-Tooth Subtype CMT2B1 in Families from North Western Africa » Ann Hum Genet (2008).

Khoury, M., Escriou, V., Courties, G., Galy, A., Yao, R., Largeau, C., Scherman, D., Jorgensen, C. and Apparailly, F.: « Efficient suppression of murine arthritis by combined anticytokine small interfering RNA lipoplexes » Arthritis Rheum 58 (2008) 2356-2367.

Krahn, M., Beroud, C., Labelle, V., Nguyen, K., Bernard, R., Bassez, G., Figarella-Branger, D., Fernandez, C., Bouvenot, J., Richard, I., Ollagnon-Roman, E., Bevilacqua, J.A., Salvo, E., Attarian, S., Chapon, F., Pellissier, J.F., Pouget, J., Hammouda, E.H., Laforet, P., Urtizberea, J.A., Eymard, B., Leturcq, F. and Levy, N.: « Analysis of the DYSF mutational spectrum in a large cohort of patients » Hum Mutat Published Online 13 October 2008.

Lorain, S., Gross, D.A., Goyenvalle, A., Danos, O., Davoust, J. and Garcia, L.: « Transient Immunomodulation Allows Repeated Injections of AAV1 and Correction of Muscular Dystrophy in Multiple Muscles » Mol Ther 16 (2008) 541–547.

Mason, A.J., Moussaoui, W., Abdelrahman, T., Boukhari, A., Bertani, P., Marquette, A., Shooshtarizaheh, P., Moulay, G., Boehm, N., Guerold, B., Sawers, R.J., Kichler, A., Metz-Boutigue, M.H., Candofi, E., Prevost, G. and Bechinger, B.: « Structural determinants of antimicrobial and antiplasmodial activity and selectivity in histidine rich amphipathic cationic peptides » J Biol Chem Published online November 4, 2008.

Neildez-Nguyen, T.M., Parisot, A., Vignal, C., Rameau, P., Stockholm, D., Picot, J., Allo, V., Le Bec, C., Laplace, C. and Paldi, A.: « Epigenetic gene expression noise and phenotypic diversification of clonal cell populations » Differentiation 76 (2008) 33-40.

Pomel, C., Leborgne, C., Cheradame, H., Scherman, D., Kichler, A. and Guegan, P.: « Synthesis and Evaluation of Amphiphilic Poly(tetrahydrofuran-b-ethylene oxide) Copolymers for DNA Delivery into Skeletal Muscle » Pharm Res Published online 4 September 2008.

Vignaud, A., Fougerousse, F., Mouisel, E., Bertrand, C., Bonafos, B., Molgo, J., Ferry, A. and Chatonnet, A.: « Genetic ablation of acetylcholinesterase alters muscle function in mice » Chem Biol Interact 175 (2008) 129-130.

Vignaud, A., Fougerousse, F., Mouisel, E., Guerchet, N., Hourde, C., Bacou, F., Butler-Browne, G.S., Chatonnet, A. and Ferry, A.: « Genetic inactivation of acetylcholinesterase causes functional and structural impairment of mouse soleus muscles » Cell Tissue Res 333 (2008) 289-296.

2007

Attali, M., Stetsyuk, V., Basmaciogullari, A., Aiello, V., Zanta-Boussif, M.A., Duvillie, B. and Scharfmann, R.: « Control of beta-cell differentiation by the pancreatic mesenchyme » Diabetes 56 (2007) 1248-58.

Bartoli, M., Poupiot, J., Vulin, A., Fougerousse, F., Arandel, L., Daniele, N., Roudaut, C., Noulet, F., Garcia, L., Danos, O. and Richard, I.: « AAV-mediated delivery of a mutated myostatin propeptide ameliorates calpain 3 but not alpha-sarcoglycan deficiency » Gene Ther 14 (2007) 733–740.

Benchaouir, R., Meregalli, M., Farini, A., D'Antona, G., Belicchi, M., Goyenvalle, A., Battistelli, M., Bresolin, N., Bottinelli, R., Garcia, L. and Torrente, Y.: « Restoration of Human Dystrophin Following Transplantation of Exon-Skipping-Engineered DMD Patient Stem Cells into Dystrophic Mice » Cell Stem Cell 1 (2007) 646.

Benchaouir, R., Picot, J., Greppo, N., Rameau, P., Stockholm, D., Garcia, L., Paldi, A. and Laplace-Builhe, C.: « Combination of quantification and observation methods for study of ”side population” cells in their “in vitro” microenvironment. » Cytometry A 71 (2007) 251-7.

Beroud, C., Tuffery-Giraud, S., Matsuo, M., Hamroun, D., Humbertclaude, W., Monnier, N., Moizard, M.P., Voelckel, M.A., Calemard, L.M., Boisseau, P., Blayau, M., Philippe, C., Cossee, M., Pages, M., Rivier, F., Danos, O., Garcia, L. and Claustres, M.: « Multiexon skipping leading to an artificial DMD protein lacking amino acids from exons 45 through 55 could rescue up to 63 % of patients with Duchenne muscular dystrophy » Hum Mutat 28 (2007) 196-202.

Brissault, B., Kichler, A., Leborgne, C., Jarroux, N., Cheradame, H. and Guis, C.: « Amphiphilic Poly[(propylene glycol)-block-(2-methyl-2-oxazoline)] Copolymers for Gene Transfer in Skeletal Muscle » ChemMedChem 2 (2007) 1202-1207.

Carmignac, V., Salih, M.A., Quijano-Roy, S., Marchand, S., Al Rayess, M.M., Mukhtar, M.M., Urtizberea, J.A., Labeit, S., Guicheney, P., Leturcq, F., Gautel, M., Fardeau, M., Campbell, K.P.,Richard, I., Estournet, B. and Ferreiro, A.: « C-terminal titin deletions cause a novel early-onset myopathy with fatal cardiomyopathy » Ann Neurol 61 (2007) 340-351.

Charrier, S., Dupre, L., Scaramuzza, S., Jeanson-Leh, L., Blundell, M.P., Danos, O., Cattaneo, F., Aiuti, A., Eckenberg, R., Thrasher, A.J., Roncarolo, M.G. and Galy, A.: « Lentiviral vectors targeting WASp expression to hematopoietic cells, efficiently transduce and correct cells from WAS patients » Gene Ther 14 (2007) 415-28.

Coecke, S., Balls, M., Bowe, G., Davis, J., Gstraunthaler, G., Hartung, T., Hay, R., Price, A.,Merten, O.-W., Stokes, W., Schlechtman, L. and Stacey, G.: « Guidance on Good Cell Culture Practice. A report of the second ECVAM task force on Good Cell Culture Practice – Proceedings of the 19th ESACT Meeting, Harrogate, UK, June 5-8, 2005 » In: Smith, R. (Ed.), Cell Technology for Cell Products. Springer, Dordrecht, 2007, pp. 313-315.

Daniele, N., Richard, I. and Bartoli, M.: « Ins and outs of therapy in limb girdle muscular dystrophies » Int J Biochem Cell Biol 39 (2007) 1608-24.

Deburgrave, N., Daoud, F., Llense, S., Barbot, J.C., Recan, D., Peccate, C., Burghes, A.H.M., Beroud, C., Garcia, L., Kaplan, J., Chelly, J. and Leturcq, F.: « Protein, and mRNABased phenotype-genotype correlations in DMD/DMD with point mutations and molecular basis for BMD with nonsense and frameshift mutations in the DMD gene » Hum Mutat 28 (2007) 183-195.

Delague, V., Jacquier, A., Hamadouche, T., Poitelon, Y., Baudot, C., Boccaccio, I., Chouery, E., Chaouch, M., Kassouri, N., Jabbour, R., Grid, D., Megarbane, A., Haase, G. and Levy, N.: « Mutations in FGD4 encoding the Rho GDP/GTP exchange factor FRABIN cause autosomal recessive Charcot-Marie-Tooth type 4H » Am J Hum Genet 81 (2007) 1-16.

Delenda, C., Chillon, M., Douar, A.M. and Merten, O.-W.: « Cells for gene therapy and vector production » In: Pörtner, R. (Ed.), Animal Cell Biotechnology: Methods and Protocols. Humana Press, Totowa, 2007, pp. 23-91.

Elleuch, N., Bouslam, N., Hanein, S., Lossos, A., Hamri, A., Klebe, S., Meiner, V., Birouk, N., Lerer, I., Grid, D., Bacq, D., Tazir, M., Zelenika, D., Argov, Z., Durr, A., Yahyaoui, M., Benomar, A., Brice, A. and Stevanin, G.: « Refinement of the SPG15 candidate interval and phenotypic heterogeneity in three large Arab families » Neurogenetics (2007).

Faure, O., Graff-Dubois, S., Alves, P.M., Cornet, S., Duffour, M.T., Scardino, A., Gross, D.A., Miconnet, I., Salcedo, M., Chouaib, S., Lemonnier, F.A., Abastado, J.P. and Kosmatopoulos, K.: « Induction of multiple CD8+ T cell responses against the inducible Hsp70 employing an Hsp70 oligoepitope peptide » Oncol Rep 17 (2007) 679-85.

Fougerousse, F., Bartoli, M., Poupiot, J., Arandel, L., Durand, M., Guerchet, N., Gicquel, E., Danos, O. and Richard, I.: « Phenotypic Correction of alpha-Sarcoglycan Deficiency by Intra-arterial Injection of a Muscle-specific Serotype 1 rAAV Vector » Mol Ther 15 (2007) 53-61.

Griggs, R., Vihola, A., Hackman, P., Talvinen, K., Haravuori, H., Faulkner, G., Eymard, B.,Richard, I., Selcen, D., Engel, A., Carpen, O. and Udd, B.: « Zaspopathy in a large classic late-onset distal myopathy family » Brain 130 (2007) 1477-84.

Israeli, D., Ziaei, S., Gjata, B., Benchaouir, R., Rameau, P., Marais, T., Fukada, S.I., Segawa, M., Yamamoto, H., Gonin, P., Danos, O. and Garcia, L.: « Expression of mdr1 is required for efficient long term regeneration of dystrophic muscle » Exp Cell Res 313 (2007) 2438-50.

Jaiswal, J.K., Marlow, G., Summerill, G., Mahjneh, I., Mueller, S., Hill, M., Miyake, K., Haase, H., Anderson, L.V., Richard, I., Kiuru-Enari, S., McNeil, P.L., Simon, S.M. and Bashir, R.: « Patients with a non-dysferlin Miyoshi myopathy have a novel membrane repair defect » Traffic 8 (2007) 77-88.

Jeanson-Leh, L., Blondeau, J. and Galy, A.: « Optimization of short hairpin RNA for lentiviral-mediated RNAi against WAS » Biochem Biophys Res Commun 362 (2007) Pages 498-503.

Kichler, A., Leborgne, C., Danos, O. and Bechinger, B.: « Characterization of the gene transfer process mediated by histidine-rich peptides » J Mol Med 85 (2007) 191-201.

Klebe, S., Durr, A., Bouslam, N., Grid, D., Paternotte, C., Depienne, C., Hanein, S., Bouhouche, A., Elleuch, N., Azzedine, H., Poea-Guyon, S., Forlani, S., Denis, E., Charon, C., Hazan, J., Brice, A. and Stevanin, G.: « Spastic paraplegia 5: Locus refinement, candidate gene analysis and clinical description » Am J Med Genet B Neuropsychiatr Genet (2007).

Lesueur, F., Bouadjar, B., Lefevre, C., Jobard, F., Audebert, S., Lakhdar, H., Martin, L., Tadini, G., Karaduman, A., Emre, S., Saker, S., Lathrop, M. and Fischer, J.: « Novel mutations in ALOX12B in patients with autosomal recessive congenital ichthyosis and evidence for genetic heterogeneity on chromosome 17p13 » J Invest Dermatol 127 (2007) 829-34.

Lesueur, F., Lefevre, C., Has, C., Guilloud-Bataille, M., Oudot, T., Mahe, E., Lahfa, M., Mansouri, S., Mosharraf-Olmolk, H., Sobel, E., Heath, S., Lathrop, M., Dizier, M.H., Prud'Homme, J.F. and Fischer, J.: « Confirmation of psoriasis susceptibility loci on chromosome 6p21 and 20p13 in French families » J Invest Dermatol 127 (2007) 1403-9.

Lesueur, F., Oudot, T., Heath, S., Foglio, M., Lathrop, M., Prud'homme, J.F. and Fischer, J.: « ADAM33, a New Candidate for Psoriasis Susceptibility » PLoS ONE 2 (2007) e906.

Mars, L.T., Bauer, J., Gross, D.A., Bucciarelli, F., Firat, H., Hudrisier, D., Lemonnier, F., Kosmatopoulos, K. and Liblau, R.S.: « CD8 T Cell Responses to Myelin Oligodendrocyte Glycoprotein-Derived Peptides in Humanized HLA-A*0201-Transgenic Mice » J Immunol 179 (2007) 5090-8.

Mason, A.J., Bechinger, B. and Kichler, A.: « Rational design of vector and antibiotic peptides using solid-state NMR. » Mini Rev Med Chem 7 (2007) 491-7.

Mason, A.J., Bertani, P., Moulay, G., Marquette, A., Perrone, B., Drake, A.F., Kichler, A. and Bechinger, B.: « Membrane Interaction of Chrysophsin-1, a Histidine-Rich Antimicrobial Peptide from Red Sea Bream » Biochemistry (2007).

Mason, A.J., Leborgne, C., Moulay, G., Martinez, A., Danos, O., Bechinger, B. and Kichler, A.: « Optimising histidine rich peptides for efficient DNA delivery in the presence of serum » J Control Release 118 (2007) 95-104.

Masurier, C., Boutin, S., Veron, P., Bernard, J., Danos, O. and Davoust, J.: « Enhanced lentiviral transduction of monocyte-derived dendritic cells in presence of conditioned medium from dying monocytes » Hum Gene Ther? (2007) 161 -170.

Maystadt, I., Rezsöhazy, R., Barkats, M., Duque, S., Vannuffel, P., Remacle, S., Lambert, B., Najimi, M., Sokal, E., Munnich, A., Viollet, L. and Verellen-Dumoulin, C.: « The Nuclear Factor B–Activator Gene PLEKHG5 Is Mutated in a Form of Autosomal Recessive Lower Motor Neuron Disease with Childhood Onset » Am J Hum Genet 81 (2007) 67-76.

Milic, A., Daniele, N., Lochmuller, H., Mora, M., Comi, G.P., Moggio, M., Noulet, F., Walter, M.C., Morandi, L., Poupiot, J., Roudaut, C., Bittner, R.E., Bartoli, M. and Richard, I.: « A third of LGMD2A biopsies have normal calpain 3 proteolytic activity as determined by an in vitro assay » Neuromuscul Disord 17 (2007) 148-56.

Paldi, A.: « Expression stochastique des gènes » Génétiquement indéterminé: le vivant auto-organisé. Editions QUAE – INRA, 2007.

Prongidi-Fix, L., Sugawara, M., Bertani, P., Raya, J., Leborgne, C., Kichler, A. and Bechinger, B.: « Self-Promoted Cellular Uptake of Peptide/DNA Transfection Complexes » Biochemistry 46 (2007) 11253-62.

Quenneville, S.P., Chapdelaine, P., Skuk, D., Paradis, M., Goulet, M., Rousseau, J., Xiao, X.,Garcia, L. and Tremblay, J.P.: « Autologous transplantation of muscle precursor cells modified with a lentivirus for muscular dystrophy: human cells and primate models » Mol Ther 15 (2007) 431-8.

Rivière, C. and Douar, A.M.: « Current challenges in AAV systemic delivery in humans. in Virus Expression Vectors » In: Hefferon, K.L. (Ed.), Virus expression vectors. Transworld Research Network, 2007, pp. Chapt 2.

Robin, P., Fritsch, L., Philipot, O., Svinartchouk, F. and Ait-Si-Ali, S.: « Post-translational modifications of histones H3 and H4 associated with the histone methyltransferases Suv39h1 and G9a » Genome Biol 8 (2007) R270.

Stevanin, G., Paternotte, C., Coutinho, P., Klebe, S., Elleuch, N., Loureiro, J.L., Denis, E., Cruz, V.T., Durr, A., Prud'homme, J.F., Weissenbach, J., Brice, A. and Hazan, J.: « A new locus for autosomal recessive spastic paraplegia (SPG32) on chromosome 14q12-q21 » Neurology 68 (2007) 1837-40.

Stevanin, G., Santorelli, F.M., Azzedine, H., Coutinho, P., Chomilier, J., Denora, P.S., Martin, E., Ouvrard-Hernandez, A.M., Tessa, A., Bouslam, N., Lossos, A., Charles, P., Loureiro, J.L., Elleuch, N., Confavreux, C., Cruz, V.T., Ruberg, M., Leguern, E., Grid, D., Tazir, M., Fontaine, B., Filla, A., Bertini, E., Durr, A. and Brice, A.: « Mutations in SPG11, encoding spatacsin, are a major cause of spastic paraplegia with thin corpus callosum » Nat Genet 39 (2007) 366-72.

Stockholm, D., Benchaouir, R., Picot, J., Rameau, P., Neildez, T.M.A., Landini, G., Laplace-Builhe, C. and Paldi, A.: « The origin of phenotypic heterogeneity in a clonal cell population in vitro » PLoS ONE 2 (2007) e394.

Tang, Y., Liu, X., Zoltoski, R.K., Novak, L.A., Herrera, R.A., Richard, I., Kuszak, J.R. and Kumar, N.M.: « Age-Related Cataracts in {alpha}3Cx46-Knockout Mice Are Dependent on a Calpain 3 Isoform » Invest Ophthalmol Vis Sci 48 (2007) 2685-94.

Vallat, J.M., Magy, L., Lagrange, E., Sturtz, F., Magdelaine, C., Grid, D. and Tazir, M.: « Diagnostic value of ultrastructural nerve examination in Charcot-Marie-Tooth disease: two CMT 1B cases with pseudo-recessive inheritance » Acta Neuropathol (Berl) 113 (2007) 443-9.

Vassilopoulos, S., Brocard, J., Garcia, L., Marty, I. and Bouron, A.: « Retrograde regulation of store-operated calcium channels by the ryanodine receptor-associated protein triadin 95 in rat skeletal myotubes » Cell Calcium 41 (2007) 179-185.

Veron, P., Allo, V., Riviere, C., Bernard, J., Douar, A.M. and Masurier, C.: « Major subsets of human dendritic cells are efficiently transduced using self-complementary adeno-associated viral vectors 1 and 2 » J Virol 81 (2007) 5385-5394.

2006

Aranyi, T. and Paldi, A.: « The constant variation: DNA methylation changes during preimplantation development » FEBS Lett 580 (2006) 6521-6.

Azzedine, H., Ravise, N., Verny, C., Gabreels-Festen, A., Lammens, M., Grid, D., Vallat, J.M., Durosier, G., Senderek, J., Nouioua, S., Hamadouche, T., Bouhouche, A., Guilbot, A., Stendel, C., Ruberg, M., Brice, A., Birouk, N., Dubourg, O., Tazir, M. and LeGuern, E.: « Spine deformities in Charcot-Marie-Tooth 4C caused by SH3TC2 gene mutations » Neurology 67 (2006) 602-6.

Balci, B., Aurino, S., Haliloglu, G., Talim, B., Erdem, S., Akcoren, Z., Tan, E., Caglar, M., Richard, I., Nigro, V., Topaloglu, H. and Dincer, P.: « Calpain-3 mutations in Turkey » Eur J Pediatr 165 (2006) 293-8.

Barde, I., Zanta-Boussif, M.A., Paisant, S., Leboeuf, M., Rameau, P., Delenda, C. and Danos, O.: « Efficient control of gene expression in the hematopoietic system using a single Tet-on inducible lentiviral vector » Mol Ther 13 (2006) 382-90.

Bartoli, M., Bourg, N., Stockholm, D., Raynaud, F., Delevaque, A., Han, Y., Borel, P., Seddik, K., Armande, N. and Richard, I.: « A mice model for monitoring calpain activity in physiological and pathological conditions » J Biol Chem 281 (2006) 39672-80.

Bartoli, M., Roudaut, C., Martin, S., Fougerousse, F., Suel, L., Poupiot, J., Gicquel, E., Noulet, F., Danos, O. and Richard, I.: « Safety and Efficacy of AAV-Mediated Calpain 3 Gene Transfer in a Mouse Model of Limb-Girdle Muscular Dystrophy Type 2A » Mol Ther 13 (2006) 250-259.

Beroud, C., Tuffery-Giraud, S., Matsuo, M., Hamroun, D., Humbertclaude, V., Monnier, N., Moizard, M.P., Voelckel, M.A., Calemard, L.M., Boisseau, P., Blayau, M., Philippe, C., Cossee, M., Pages, M., Rivier, F., Danos, O., Garcia, L. and Claustres, M.: « Multiexon skipping leading to an artificial DMD protein lacking amino acids from exons 45 through 55 could rescue up to 63% of patients with Duchenne muscular dystrophy » Hum Mutat 28 (2006) 196 – 202.

Blewitt, M.E., Vickaryous, N.K., Paldi, A., Koseki, H. and Whitelaw, E.: « Dynamic reprogramming of DNA methylation at an epigenetically sensitive allele in mice » PLoS Genet 2 (2006) e49.

Brissault, B., Kichler, A., Leborgne, C., Danos, O., Cheradame, H., Gau, J., Auvray, L. and Guis, C.: « Synthesis, characterization, and gene transfer application of poly(ethylene glycol-b-ethylenimine) with high molar mass polyamine block. » Biomacromolecules 7 (2006) 2863-2870.

Brissault, B., Leborgne, C., Guis, C., Danos, O., Cheradame, H. and Kichler, A.: « Linear topology confers in vivo gene transfer activity to polyethylenimines » Bioconjug Chem 17 (2006) 759-65.

Burckbuchler, V., Wintgens, V., Lecomte, S., Percot, A., Leborgne, C., Danos, O., Kichler, A.and Amiel, C.: « DNA compaction into new DNA vectors based on cyclodextrin polymer: SERS characterization » Biopolymers 81 (2006) 360-70.

Cabarrocas, J., Cassan, C., Magnusson, F., Piaggio, E., Mars, L., Derbinski, J., Kyewski, B.,Gross, D.A., Salomon, B.L., Khazaie, K., Saoudi, A. and Liblau, R.S.: « Foxp3+ CD25+ regulatory T cells specific for a neo-self-antigen develop at the double-positive thymic stage » Proc Natl Acad Sci U S A 103 (2006) 8453-8.

Capron, C., Lecluse, Y., Kaushik, A.L., Foudi, A., Lacout, C., Sekkai, D., Godin, I., Albagli, O., Poullion, I., Svinartchouk, F., Schanze, E., Vainchenker, W., Sablitzky, F., Bennaceur-Griscelli, A. and Dumenil, D.: « The SCL relative LYL-1 is required for fetal and adult hematopoietic stem cell function and B-cell differentiation » Blood 107 (2006) 4678-86.

Carmo, M., Faria, T.Q., Falk, H., Coroadinha, A.S., Teixeira, M., Merten, O.-W., Gény-Fiamma, C., Alves, P.M., Danos, O., Panet, A., Carrondo, M.J.T. and Cruz, P.E.: « Relationship between retroviral vector membrane and vector stability » J Gen Virol 87 (2006) 1349-56.

Cheradame, H., Brissault, B., Guis, C., Guégan, P., Pomel, C., Kichler, A., Gau, J. and Auvray, L.: « Improvement of synthetic vectors for gene therapy using ring-opening cationic polymerization » Macromolecular Symposia 240 (2006) 166-177.

Coroadinha, A.S., Alves, P.M., Santos, S.S., Cruz, P.E., Merten, O.-W. and Carrondo, M.J.: « Retrovirus producer cell line metabolism: implications on viral productivity » Appl Microbiol Biotechnol 72 (2006) 1125-35.

Coroadinha, A.S., Ribeiro, J., Roldao, A., Cruz, P.E., Alves, P.M., Merten, O.-W. and Carrondo, M.J.: « Effect of medium sugar source on the production of retroviral vectors for gene therapy » Biotechnol Bioeng 94 (2006) 24-36.

Danos, O. and Svinartchouk, F.: « Dialysis-assisted two-dimensional gel electrophoresis » Electrophoresis 27 (2006) 3475 – 3479.

Deburgrave, N., Daoud, F., Llense, S., Barbot, J.C., Recan, D., Peccate, C., Burghes, A.H., Beroud, C., Garcia, L., Kaplan, J.C., Chelly, J. and Leturcq, F.: « Protein- and mRNA-based phenotype-genotype correlations in DMD/BMD with point mutations and molecular basis for BMD with nonsense and frameshift mutations in the DMD gene » Hum Mutat 28 (2006) 183 – 195.

Dubourg, O., Azzedine, H., Verny, C., Durosier, G., Birouk, N., Gouider, R., Salih, M., Bouhouche, A., Thiam, A., Grid, D., Mayer, M., Ruberg, M., Tazir, M., Brice, A. and LeGuern, E.: « Autosomal-recessive forms of demyelinating Charcot-Marie-Tooth disease » Neuromolecular Med 8 (2006) 75-86.

Duguez, S., Bartoli, M. and Richard, I.: « Calpain 3: a key regulator of the sarcomere? » Febs J 273 (2006) 3427-36.

Elleuch, N., Depienne, C., Benomar, A., Hernandez, A.M., Ferrer, X., Fontaine, B., Grid, D., Tallaksen, C.M., Zemmouri, R., Stevanin, G., Durr, A. and Brice, A.: « Mutation analysis of the paraplegin gene (SPG7) in patients with hereditary spastic paraplegia » Neurology 66 (2006) 654-9.

Etzel, C.J., Chen, W.V., Shepard, N., Jawaheer, D., Cornelis, F., Seldin, M.F., Gregersen, P.K. and Amos, C.I.: « Genome-wide meta-analysis for rheumatoid arthritis » Human Genet 119 (2006) 634-641.

Gross, D.A., Chappert, P., Leboeuf, M., Monteilhet, V., Van Wittenberghe, L., Danos, O. and Davoust, J.: « Simple conditioning with mono-specific CD4+CD25+ regulatory T cells for bone marrow engraftment and tolerance to multiple gene products » Blood 108 (2006) 1841-1848.

Hempelmann, A., Taylor, K.P., Heils, A., Lorenz, S., Prud'homme, J.F., Nabbout, R., Dulac, O., Rudolf, G., Zara, F., Bianchi, A., Robinson, R., Gardiner, R.M., Covanis, A., Lindhout, D., Stephani, U., Elger, C.E., Weber, Y.G., Lerche, H., Nurnberg, P., Kron, K.L., Scheffer, I.E., Mulley, J.C., Berkovic, S.F. and Sander, T.: « Exploration of the genetic architecture of idiopathic generalized epilepsies » Epilepsia 47 (2006) 1682-90.

Izmiryan, A., Cheraud, Y., Khanamiryan, L., Leterrier, J.F., Federici, T., Peltekian, E., Moura-Neto, V., Paulin, D., Li, Z. and Xue, Z.G.: « Different expression of synemin isoforms in glia and neurons during nervous system development » Glia 54 (2006) 204-213.

Kichler, A., Mason, A.J. and Bechinger, B.: « Cationic amphipathic histidine-rich peptides for gene delivery » Biochim Biophys Acta 1758 (2006) 301-7.

Krahn, M., Bernard, R., Pecheux, C., Hammouda, E.H., Eymard, B., De Munain, A.L., Cobo, A.M., Romero, N., Urtizberea, A., Leturcq, F. and Levy, N.: « Screening of the CAPN3 gene in patients with possible LGMD2A » Clin Genet 69 (2006) 444-449.

Lagresle-Peyrou, C., Yates, F., Malassis-Seris, M., Hue, C., Morillon, E., Garrigue, A., Liu, A., Hajdari, P., Stockholm, D., Danos, O., Lemercier, B., Gougeon, M.L., Rieux-Laucat, F., de Villartay, J.P., Fischer, A. and Cavazzana-Calvo, M.: « Long term immune reconstitution in RAG-1 deficient mice treated by retroviral gene therapy: a balance between efficiency and toxicity » Blood 107 (2006) 63-72.

Le Bec, C. and Douar, A.M.: « Gene Therapy Progress and Prospects – Vectorology: design and production of expression cassettes in AAV vectors » Gene Ther 13 (2006) 805-13.

Lefevre, C., Bouadjar, B., Ferrand, V., Tadini, G., Megarbane, A., Lathrop, M., Prud'homme, J.F. and Fischer, J.: « Mutations in a new cytochrome P450 gene in lamellar ichthyosis type 3 » Hum Mol Genet 15 (2006) 767-76.

Mantovani, J., Holic, N., Martinez, K., Danos, O. and Perea, J.: « A high throughput method for genome-wide analysis of retroviral integration » Nucleic Acids Res 34 (2006) e134.

Marquis, J., Paillard, L., Audic, Y., Cosson, B., Danos, O., Le Bec, C. and Osborne, H.B.: « CUG-BP1/CELF1 requires UGU-rich sequences for high affinity binding » Biochem J 400 (2006) 291-301.

Mas, A., Montane, J., Anguela, X.M., Munoz, S., Douar, A.M., Riu, E., Otaegui, P. and Bosch, F.: « Reversal of type 1 diabetes by engineering a glucose sensor in skeletal muscle » Diabetes 55 (2006) 1546-53.

Mason, A.J., Gasnier, C., Kichler, A., Prévost, G., Aunis, D., Metz-Boutigue, M.-H. and Bechinger, B.: « Designed histidine-rich peptides show pH dependent antibiotic action against pathogenic bacteria » Antimicrob Agents Chemother. 2006 (2006) 3305-3311.

Mason, A.J., Martinez, A., Glaubitz, C., Danos, O., Kichler, A. and Bechinger, B.: « The antibiotic and DNA transfecting peptide LAH4 selectively associates with, and disorders, anionic lipids in mixed membranes » FASEB Journal 20 (2006) 320-2.

Merten, O.W.: « Introduction to animal cell culture technology – past, present and future » Cytotechnology 50 (2006) 1-7.

Olivier, A., Jeanson-Leh, L., Bouma, G., Compagno, D., Blondeau, J., Seye, K., Charrier, S., Burns, S., Thrasher, A., Danos, O., Vainchenker, W. and Galy, A.: « A partial down-regulation of WASP is sufficient to inhibit podosome formation in dendritic cells » Mol Ther 13 (2006) 729-37.

Olivier, A., Lauret, E., Gonin, P. and Galy, A.: « The Notch ligand delta-1 is a hematopoietic development co-factor for plasmacytoid dendritic cells » Blood 107 (2006) 2694-701.

Paldi, A.: « Empreinte génomique » Métabolismes Hormones Diabètes et Nutrition 10 (2006) 6-9.

Parouchev, A., Nguyen, T.H., Dagher, I., Mainot, S., Groyer-Picard, M.T., Branger, J., Gonin, P., Di Santo, J., Franco, D., Gras, G. and Weber, A.: « Efficient ex vivo gene transfer into non-human primate hepatocytes using HIV-1 derived lentiviral vectors » J Hepatol 45 (2006) 99-107.

Partridge, T.: « Disciplining the stem cell into myogenesis » N Engl J Med 354 (2006) 1844-1845.

Rameau, P. and Laplace-Builhe, C.: « Le tri à haute vitesse » In: Ronot, X., Grunwald, D., Mayol, J.F. and Boutonnat, J. (Eds.), La cytométrie en flux: un besoin circonstanciel en biologie et en médecine. Technique & Documentation, Paris, 2006.

Richard, I.: « Limb-girdle muscular dystrophies » In: Ganten, D.R., K. (Ed.), Encyclopedic Reference of Genomics and Proteomics. Springer-Verlag, 2006.

Riviere, C., Danos, O. and Douar, A.M.: « Long term expression and repeated administration of AAV type 1, 2 and 5 vectors in skeletal muscle of immunocompetent adult mice » Gene Ther 13 (2006) 1300 -1308.

Rowe, H.M., Lopes, L., Ikeda, Y., Bailey, R., Barde, I., Zenke, M., Chain, B.M. and Collins, M.K.: « Immunization with a Lentiviral Vector Stimulates both CD4 and CD8 T Cell Responses to an Ovalbumin Transgene » Mol Ther 13 (2006) 310-9.

Schucht, R., Coroadinha, A.S., Zanta-Boussif, M.A., Verhoeyen, E., Carrondo, M.J., Hauser, H. and Wirth, D.: « A new generation of retroviral producer cells: predictable and stable virus production by flp-mediated site-specific integration of retroviral vectors » Mol Ther 14 (2006) 285-92.

Smet-Nocca, C., Paldi, A. and Benecke, A.: « De l'épigénomique à l'émergence morphogénétique » In: Bourgine, P. and Lesne, A. (Eds.), Morphogenèse. L'origine des formes. Belin, Paris, 2006.

Spirito, F., Capt, A., Del Rio, M., Larcher, F., Guaguere, E., Danos, O. and Meneguzzi, G.: « Sustained phenotypic reversion of junctional epidermolysis bullosa dog keratinocytes: Establishment of an immunocompetent animal model for cutaneous gene therapy » Biochem Biophys Res Commun 339 (2006) 769-778.

Striano, P., Lispi, M.L., Gennaro, E., Madia, F., Traverso, M., Bordo, L., Aridon, P., Boneschi, F.M., Barone, B., dalla Bernardina, B., Bianchi, A., Capovilla, G., De Marco, P., Dulac, O., Gaggero, R., Gambardella, A., Nabbout, R., Prud'homme, J.F., Day, R., Vanadia, F., Vecchi, M., Veggiotti, P., Vigevano, F., Viri, M., Minetti, C. and Zara, F.: « Linkage analysis and disease models in benign familial infantile seizures: A study of 16 families » Epilepsia 47 (2006) 1029-1034.

Striano, P., Lispi, M.L., Gennaro, E., Madia, F., Traverso, M., Bordo, L., Aridon, P., Boneschi, F.M., Barone, B., dalla Bernardina, B., Bianchi, A., Capovilla, G., De Marco, P., Dulac, O., Gaggero, R., Gambardella, A., Nabbout, R., Prud'homme, J.F., Day, R., Vanadia, F., Vecchi, M., Veggiotti, P., Vigevano, F., Viri, M., Minetti, C. and Zara, F.: « Linkage analysis and disease models in benign familial infantile seizures: a study of 16 families » Epilepsia 47 (2006) 1029-34.

t'Hoen, P.A., van der Wees, C.G., Aartsma-Rus, A., Turk, R., Goyenvalle, A., Danos, O.,Garcia, L., van Ommen, G.J., den Dunnen, J.T. and van Deutekom, J.C.: « Gene expression profiling to monitor therapeutic and adverse effects of antisense therapies for Duchenne muscular dystrophy » Pharmacogenomics 7 (2006) 281-97.

Veron, P., Boutin, S., Bernard, J., Danos, O., Davoust, J. and Masurier, C.: « Efficient transduction of monocyte- and CD34(+)- derived Langerhans cells with lentiviral vectors in the absence of phenotypic and functional maturation » J Gene Med 8 (2006) 951 – 961.

Warnock, J.N., Merten, O.-W. and Al, R.M.: « Cell culture processes for the production of viral vectors for gene therapy purposes » Cytotechnology 50 (2006) 141.

2005

Abecasis, G., Allen, M., Barker, J., Burden, D., Capon, F., Christophers, E., Elder, J.T., Fischer, J., Gudjonsson, J.E., Huffmeier, U., Jenisch, S., Karason, A., Kere, J., Nair, R.P., Novelli, G., Prud'homme, J.F., Qin, Z.H.S., Samuelsson, L., Sanchez, F., Saarialho-Kere, U., Stahle, M., Stuart, P., Tillman, D., Traupe, H., Trembath, R., Valdimarsson, H., Veal, C., Voorhees, J.J. and Weichenthal, M.: « Fine mapping of the psoriasis susceptibility gene PSORS1: A reassessment of risk associated with a putative risk haplotype lacking HLA-Cw6 » J Invest Dermatol 124 (2005) 921-930.

Apparailly, F., Khoury, M., Vervoordeldonk, M.J.B., Adriaansen, J., Gicquel, E., Perez, N., Riviere, C., Louis-Plence, P., Noel, D., Danos, O., Douar, A.M., Tak, P.P. and Jorgensen, C.: « Adeno-associated virus pseudotype 5 vector improves gene transfer in arthritic joints » Hum Gene Ther 16 (2005) 426-434.

Baconnais, S., Delavoie, F., Zahm, J.M., Milliot, M., Terryn, C., Castillon, N., Banchet, V., Michel, J., Danos, O., Merten, M., Chinet, T., Zierold, K., Bonnet, N., Puchelle, E. and Balossier, G.: « Abnormal ion content, hydration and granule expansion of the secretory granules from cystic fibrosis airway glandular cells » Exp Cell Res 309 (2005) 296-304.

Bagnis, C., Merten, O.-W. and Mezzina, M.: « Advanced methods for industrial production, purification, and characterization of gene vectors » Gene Ther 12 (2005) 1-177.

Bartoli, M. and Richard, I.: « Calpains and muscle wasting » Int J Biochem Cell Biol 37 (2005) 2115-2133.

Bartoli, M., Poupiot, J., Goyenvalle, A., Perez, N., Garcia, L., Danos, O. and Richard, I.: « Noninvasive monitoring of therapeutic gene transfer in animal models of muscular dystrophies » Gene Ther 13 (2005) 20-28.

Berthebaud, M., Riviere, C., Jarrier, P., Foudi, A., Zhang, Y., Compagno, D., Galy, A., Vainchenker, W. and Louache, F.: « RGS16 is a negative regulator of SDF-1-CXCR4 signaling in megakaryocytes » Blood 106 (2005) 2962-8.

Bertho, N., Adamski, H., Toujas, L., Debove, M., Davoust, J. and Quillien, V.: « Efficient migration of dendritic cells towards lymph node chemokines and induction of Th1 responses require maturation stimulus and apoptotic cells interaction » Blood 106 (2005) 1734-1741.

Borgnon, J., Djamouri, F., Lorand, I., Di Rico, V., Loux, N., Pages, J.C., Franco, D., Capron, F. and Weber, A.: « Follistatin allows efficient retroviral-mediated gene transfer into rat liver » Biochem Biophys Res Commun 328 (2005) 937-943.

Charrier, S., Stockholm, D., Seye, K., Opolon, P., Taveau, M., Gross, D.A., Bucher-Laurent, S., Delenda, C., Vainchenker, W., Danos, O. and Galy, A.: « A lentiviral vector encoding the human Wiskott-Aldrich syndrome protein corrects immune and cytoskeletal defects in WASP knockout mice » Gene Ther 12 (2005) 597-606.

Coecke, S., Balls, M., Bowe, G., Davis, J., Gstraunthaler, G., Hartung, T., Hay, R., Merten, O.-W., Price, A., Schechtman, L., Stacey, G. and Stokes, W.: « Guidance on Good Cell Culture Practice: A Report of the Second ECVAM Task Force on Good Cell Culture Practice » Alternatives to laboratory animals: ATLA. 33 (2005) 261-287.

Collins, C.A. and Partridge, T.A.: « Self-renewal of the adult skeletal muscle satellite cell » Cell Cycle 4 (2005) 1338-1341.

Cruz, P.E., Carmo, M., Coroadinha, A.S., Bengala, A., Goncalves, D., Teixeira, M., Merten, O.-W., Gény-Fiamma, C. and Carrondo, M.J.T.: « Retroviral vector stability: inactivation kinetics and membrane properties. » In: Godia, F. and Fussenegger, M. (Eds.), Animal cell technology meets genomics. Springer, Dordrecht, 2005, pp. 303-308.

Davoust, J., Bertho, N., Masurier, C. and Gross, D.: « Immunity and tolerance induction in gene therapy » In: Dunn, M.J., Jorde, L.B., Little, P.F.R. and Subramaniam, S. (Eds.), Encyclopedia of genetics, genomics, proteomics and bioinformatics. John Wiley & Sons, Ltd., New York, 2005, pp. 817-25.

De Sandre-Giovannoli, A., Delague, V., Hamadouche, T., Chaouch, M., Krahn, M., Boccaccio, I., Maisonobe, T., Chouery, E., Jabbour, R., Atweh, S., Grid, D., Megarbane, A. and Levy, N.: « Homozygosity mapping of autosomal recessive demyelinating Charcot-Marie-Tooth neuropathy (CMT4H) to a novel locus on chromosome 12p11.21-q13.11 » J Med Genet 42 (2005) 260-5.

Decraene, C., Benchaouir, R., Dillies, M.A., Israeli, D., Bortoli, S., Rochon, C., Rameau, P., Pitaval, A., Tronik-Le Roux, D., Danos, O., Gidrol, X., Garcia, L. and Pietu, G.: « Global transcriptional characterization of SP and MP cells from the myogenic C2C12 cell line: effect of FGF6 » Physiol Genomics 23 (2005) 132-49.

Decraene, C., Benchaouir, R., Dillies, M.A., Israeli, D., Bortoli, S., Rochon, C., Rameau, P., Pitaval, A., Tronik-Le Roux, D., Danos, O., Gidrol, X., Garcia, L. and Pietu, G.: « Global transcriptional characterization of SP and MP cells from the myogenic C2C12 cell line: effect of FGF6 » Physiological Genomics 23 (2005) 132-149.

Delenda, C. and Gaillard, C.: « Real-time quantitative PCR for the design of lentiviral vector analytical assays » Gene Ther 12 Suppl 1 (2005) S36-50.

Fleurence, E., Riviere, C., Lacaze-Masmonteil, T., Franco-Motoya, M.L., Waszak, P., Bourbon, J., Danos, O., Douar, A.M. and Delacourt, C.: « Comparative efficacy of intratracheal adeno-associated virus administration to newborn rats » Hum Gene Ther 16 (2005) 1298-306.

Gagnoux-Palacios, L., Hervouet, C., Spirito, F., Roques, S., Mezzina, M., Danos, O. and Meneguzzi, G.: « Assessment of optimal transduction of primary human skin keratinocytes by viral vectors » J Gene Med 7 (2005) 1178-86.

Gonin, P., Arandel, L., Van Wittenberghe, L., Marais, T., Perez, N. and Danos, O.: « Femoral intra-arterial injection: a tool to deliver and assess recombinant AAV constructs in rodents whole hind limb » J Gene Med 7 (2005) 782-91.

Gonin, P., Buchholz, C.J., Pallardy, M. and Mezzina, M.: « Gene therapy bio-safety: scientific and regulatory issues » Gene Ther 12 Suppl 1 (2005) S146-52.

Gonin, P.: « Entamoeba histolytica, Entamoeba dispar » In: Fuchs, J. and Podda, M. (Eds.), Encyclopedia of Medical Genomics and Proteomics. Marcel Dekker, New-York, 2005, pp. 391 – 394.

Imamura, T., Kerjean, A., Heams, T., Kupiec, J.J., Thenevin, C. and Paldi, A.: « Dynamic CpG and non-CpG methylation of the Peg1/Mest gene in the mouse oocyte and preimplantation embryo » J Biol Chem 280 (2005) 20171-5.

Israeli, D., Ziaei, S., Gonin, P. and Garcia, L.: « A proposal for the physiological significance of mdr1 and Bcrp1/Abcg2 gene expression in normal tissue regeneration and after cancer therapy » J Theor Biol 232 (2005) 41-5.

Jenny, C., Toublanc, E., Danos, O. and Merten, O.-W.: « Evaluation of a Serum-free Medium for the Production of rAAV-2 using HeLa Derived Producer Cells » Cytotech 49 (2005) 11-23.

Kichler, A., Leborgne, C. and Danos, O.: « Dilution of reporter gene with stuffer DNA does not alter the transfection efficiency of polyethylenimines » J Gene Med 7 (2005) 1459-67.

Kichler, A., Leborgne, C., Savage, P.B. and Danos, O.: « Cationic steroid antibiotics demonstrate DNA delivery properties » J Control Release 107 (2005) 174-82.

Kissenpfennig, A., Henri, S., Dubois, B., Laplace-Builhe, C., Perrin, P., Romani, N., Tripp, C.H., Douillard, P., Leserman, L., Kaiserlian, D., Saeland, S., Davoust, J. and Malissen, B.: « Dynamics and function of Langerhans cells in vivo dermal dendritic cells colonize lymph node areas distinct from slower migrating Langerhans cells » Immunity 22 (2005) 643-54.

Kocaefe, Y.C., Israeli, D., Ozguc, M., Danos, O. and Garcia, L.: « Myogenic program induction in mature fat tissue (with MyoD expression) » Exp Cell Res 308 (2005) 300-8.

Komura, E., Tonetti, C., Penard-Lacronique, V., Chagraoui, H., Lacout, C., Lecouedic, J.P., Rameau, P., Debili, N., Vainchenker, W. and Giraudier, S.: « Role for the nuclear factor kappaB pathway in transforming growth factor-beta1 production in idiopathic myelofibrosis: possible relationship with FK506 binding protein 51 overexpression » Cancer Res 65 (2005) 3281-9.

Kreij, K., Mandenius, C.F., Clemente, J.J., Cunha, A.E., Monteiro, S.M.S., Carrondo, M.J.T., Hesse, F., Bassani Molinas, M.d.l.M., Wagner, R., Merten, O.-W., Gény-Fiamma, C., Leger, W., Wiesinger-Mayr, H., Müller, D., Katinger, H., Martensson, P., Bachinger, T. and Mitrovics, J.: « On-line detection of microbial contaminations in animal cell reactor cultures using an electronic nose device » Cytotech 48 (2005) 41-58.

Kreij, K., Mandenius, C.F., Clemente, J.J., Cunha, A.E., Monteiro, S.M.S., Carrondo, M.J.T., Hesse, F., Molinas, M., Wagner, R., Merten, O.W., Geny-Katinger, C., Martensson, P., Bachinger, T. and Mitrovics, J.: « On-line detection of microbial contaminations in animal cell reactor cultures using an electronic nose device » Cytotechnology 48 (2005) 41-58.

Lange, S., Xiang, F., Yakovenko, A., Vihola, A., Hackman, P., Rostkova, E., Kristensen, J., Brandmeier, B., Franzen, G., Hedberg, B., Gunnarsson, L.G., Hughes, S.M., Marchand, S., Sejersen, T., Richard, I., Edstrom, L., Ehler, E., Udd, B. and Gautel, M.: « The kinase domain of titin controls muscle gene expression and protein turnover » Science 308 (2005) 1599-603.

Merten, O.-W., Gény-Fiamma, C. and Douar, A.M.: « Current issues in adeno-associated viral vectors production » Gene Ther 12 (2005) S51-S61.

Mezzina, M.: « The 3rd European conference and practical course on advanced gene vector technologies: training on pharmaceutical development of gene therapy products » Gene Ther 12 Suppl 1 (2005) S1-4.

Pascolo, S., Ginhoux, F., Laham, N., Walter, S., Schoor, O., Probst, J., Rohrlich, P., Obermayr, F., Fisch, P., Danos, O., Ehrlich, R., Lemonnier, F.A. and Rammensee, H.G.: « The non-classical HLA class I molecule HFE does not influence the NK-like activity contained in fresh human PBMCs and does not interact with NK cells » Int Immunol 17 (2005) 117-122.

Proust, A., Guillet, B., Pellier, I., Rachieru, P., Hoarau, C., Claeyssens, S., Leonard, C., Charrier, S., Vainchenker, W., Tchernia, G. and Delaunay, J.: « Recurrent V75M mutation within the Wiskott-Aldrich syndrome protein: description of a homozygous female patient » Eur J Haematol 75 (2005) 54-9.

Rezgui, S.S., Vassilopoulos, S., Brocard, J., Platel, J.C., Bouron, A., Arnoult, C., Oddoux, S., Garcia, L., De Waard, M. and Marty, I.: « Triadin (trisk 95) overexpression blocks excitation-contraction coupling in rat skeletal myotubes » J Biol Chem 280 (2005) 39302-8.

Richard, I.: « The genetic and molecular bases of monogenic disorders affecting proteolytic systems » J Med Genet 42 (2005) 529-39.

Rohrlich, P.S., Fazilleau, N., Ginhoux, F., Firat, H., Michel, F., Cochet, M., Laham, N., Roth, M.P., Pascolo, S., Nato, F., Coppin, H., Charneau, P., Danos, O., Acuto, O., Ehrlich, R., Kanellopoulos, J. and Lemonnier, F.A.: « Direct recognition by alphabeta cytolytic T cells of Hfe, a MHC class Ib molecule without antigen-presenting function » Proc Natl Acad Sci U S A 102 (2005) 12855-60.

Stockholm, D., Bartoli, M., Sillon, G., Bourg, N., Davoust, J. and Richard, I.: « Imaging calpain protease activity by multiphoton FRET in living mice » J Mol Biol 346 (2005) 215-222.

Tine, J.A., Firat, H., Payne, A., Russo, G., Davis, S.W., Tartaglia, J., Lemonnier, F.A., Demoyen, P.L. and Moingeon, P.: « Enhanced multiepitope-based vaccines elicit CD8(+) cytotoxic T cells against both immunodominant and cryptic epitopes » Vaccine 23 (2005) 1085-91.

Udd, B., Vihola, A., Sarparanta, J., Richard, I. and Hackman, P.: « Titinopathies and extension of the M-line mutation phenotype beyond distal myopathy and LGMD2J » Neurology 64 (2005) 636-42.

Vallat, J.M., Grid, D., Magdelaine, C., Sturtz, F., Levy, N. and Tazir, M.: « [Autosomal recessive forms of Charcot-Marie-Tooth disease] » Bull Acad Natl Med 189 (2005) 55-68; discussion 68-9.

Vallat, J.M., Tazir, M., Magdelaine, C., Sturtz, F. and Grid, D.: « Autosomal-recessive Charcot-Marie-Tooth diseases » J Neuropathol Exp Neurol 64 (2005) 363-70.

Vendome, J., Letard, S., Martin, F., Svinarchuk, F., Dubreuil, P., Auclair, C. and Le Bret, M.: « Molecular modeling of wild-type and D816V c-Kit inhibition based on ATP-competitive binding of ellipticine derivatives to tyrosine kinases » J Med Chem 48 (2005) 6194-201.

Young, J., Morbois-Trabut, L., Couzinet, B., Lascols, O., Dion, E., Béréziat, V., Fève, B., Richard, I., Capeau, J., Chanson, P. and Vigouroux, C.: « Type A insulin resistance syndrome revealing a novel lamin A mutation » Diabetes 54 (2005) 1873-1878.

2004

Beauchamp-Nicoud, A., Da Costa, L., Proust, A., Rince, P., Saker, S. and Tchernia, G.: « Postmortem diagnosis of Diamond-Blackfan anemia » J Pediatr Hematol Oncol 26 (2004) 847-8.

Benchaouir, R., Rameau, P., Decraene, C., Dreyfus, P., Israeli, D., Pietu, G., Danos, O. and Garcia, L.: « Evidence for a resident subset of cells with SP phenotype in the C2C12 myogenic line: a tool to explore muscle stem cell biology » Exp Cell Res 294 (2004) 254-68.

Bernot, A. and Weissenbach, J.: « Estimation of the extent of Synteny between Tetvaodon nigroviridis and Homo sapiens genomes » Mol Evol 59 (2004) 556-569.

Bidou, L., Hatin, I., Perez, N., Allamand, V., Panthier, J.J. and Rousset, J.P.: « Premature stop codons involved in muscular dystrophies show a broad spectrum of readthrough efficiencies in response to gentamicin treatment » Gene Ther 11 (2004) 619-27.

Borg, C., Abdelali, J., Laderach, D., Maruyama, K., Wakasugi, H., Charrier, S., Ryffel, B., Cambi, A., Figdor, C., Vainchenker, W., Galy, A., Caignard, A. and Zitvogel, L.: « NK cell activation by dendritic cells (DCs) requires the formation of a synapse leading to IL-12 polarization in DCs » Blood 104 (2004) 3267-3275.

Castillon, N., Avril-Delplanque, A., Coraux, C., Delenda, C., Peault, B., Danos, O. and Puchelle, E.: « Regeneration of a well-differentiated human airway surface epithelium by spheroid and lentivirus vector-transduced airway cells » J Gene Med 6 (2004) 846-56.

Caux, F., Selma, Z.B., Laroche, L., Prud'homme, J.F. and Fischer, J.: « CGI-58/ABHD5 gene is mutated in Dorfman-Chanarin syndrome » Am J Med Genet A 129 (2004) 214.

Danos, O. and Mezzina, M.: « The first conference-course on vectorology: training young researchers to implement gene therapy » J Gene Med 6 (2004) S1-S2.

Delaunay, J., Nouyrigat, V., Proust, A., Schischmanoff, P.O., Cynober, T., Yvart, J., Gaillard, C., Danos, O. and Tchernia, G.: « Different impacts of alleles alphaLEPRA and alphaLELY as assessed versus a novel, virtually null allele of the SPTA1 gene in trans » Br J Haematol 127 (2004) 118-22.

Delaunay, J., Nouyrigat, V., Proust, A., Schischmanoff, P.O., Cynober, T., Yvart, J., Gaillard, C., Danos, O. and Tchernia, G.: « Different impacts of alleles alpha(LEPRA) and alpha(LELY) as assessed versus a novel, virtually null allele of the SPTA1 gene in trans » Br J Haematol 127 (2004) 118-122.

Delenda, C.: « Lentiviral vectors: optimization of packaging, transduction and gene expression » J Gene Med 6 (2004) S125-38.

Demir, E., Prud'homme, J.F. and Topcu, M.: « Infantile convulsions and paroxysmal choreoathetosis in a consanguineous family » Pediatr Neurol 30 (2004) 349-53.

Dreyfus, P.A., Chretien, F., Chazaud, B., Kirova, Y., Caramelle, P., Garcia, L., Butler-Browne, G. and Gherardi, R.K.: « Adult bone marrow-derived stem cells in muscle connective tissue and satellite cell niches » Am J Pathol 164 (2004) 773-9.

Duan, M., Venail, F., Spencer, N. and Mezzina, M.: « Treatment of peripheral sensorineural hearing loss: gene therapy » Gene Ther 11 (2004) S51-S56.

Gény-Fiamma, C., Millot, L., Rocca, C., Danos, O. and Merten, O.-W.: « Optimization of the production of retroviral vectors – Influences of the sugar source » In: Kitagawa, Y., Matsuda, T. and Iijima, S. (Eds.), Animal cell technology: basic and applied aspects. Kluwer Academic Publishers, Dordrecht, 2004, pp. 89-97.

Ginhoux, F., Turbant, S., Gross, D.A., Poupiot, J., Marais, T., Lone, Y., Lemonnier, F.A., Firat, H., Perez, N., Danos, O. and Davoust, J.: « HLA-A*0201-restricted cytolytic responses to the rtTA transactivator dominant and cryptic epitopes compromise transgene expression induced by the tetracycline on system » Mol Ther 10 (2004) 279-89.

Gonin, P. and Gaillard, C.: « Gene transfer vector biodistribution: pivotal safety studies in clinical gene therapy development » Gene Ther 11 (2004) S98-S108.

Goyenvalle, A., Vulin, A., Fougerousse, F., Leturcq, F., Kaplan, J.C., Garcia, L. and Danos, O.: « [An opening in Duchenne muscular dystrophy: persistent therapeutic rescue of dystrophin by vectorized antisense mediated exon skipping in mdx mice.] » Med Sci (Paris) 20 (2004) 1163-1165.

Goyenvalle, A., Vulin, A., Fougerousse, F., Leturcq, F., Kaplan, J.C., Garcia, L. and Danos, O.: « Rescue of dystrophic muscle through U7 snRNA-mediated Exon-Skipping » Science 306 (2004) 1796-9.

Gross, D.A., Graff-Dubois, S., Opolon, P., Cornet, S.S., Alves, P., Bennaceur-Griscelli, A., Faure, O., Guillaume, P., Firat, H.H., Chouaib, S., Lemonnier, F.A., Davoust, J., Miconnet, I., Vonderheide, R.H. and Kosmatopoulos, K.: « High vaccination efficiency of low-affinity epitopes in antitumor immunotherapy » J Clin Invest 113 (2004) 425-33.

Haack, K., Cockrell, A.S., Ma, H., Israeli, D., Ho, S.N., McCown, T.J. and Kafri, T.: « Transactivator and structurally optimized inducible lentiviral vectors » Mol Ther 10 (2004) 585-596.

Israeli, D., Benchaouir, R., Ziaei, S., Rameau, P., Gruszczynski, C., Peltekian, E., Danos, O. and Garcia, L.: « FGF6 mediated expansion of a resident subset of cells with SP phenotype in the C2C12 myogenic line » J Cell Physiol 201 (2004) 409-419.

Kichler, A.: « Gene transfer with modified polyethylenimines » J Gene Med 6 (2004) S3-10.

Lefevre, C., Bouadjar, B., Karaduman, A., Jobard, F., Saker, S., Ozguc, M., Lathrop, M., Prud'homme, J.F. and Fischer, J.: « Mutations in ichthyin a new gene on chromosome 5q33 in a new form of autosomal recessive congenital ichthyosis » Hum Mol Genet 18 (2004) 18.

Lemoine, F.M., Mesel-Lemoine, M., Cherai, M., Gallot, G., Vie, H., Leclercq, V., Trebeden-Negre, H., Mammes, O., Boyer, O., Noguiez-Hellin, P. and Klatzmann, D.: « Efficient transduction and selection of human T-lymphocytes with bicistronic Thy1/HSV1-TK retroviral vector produced by a human packaging cell line » J Gene Med 6 (2004) 374-86.

Martineau, Y., Le Bec, C., Monbrun, L., Allo, V., Chiu, I.M., Danos, O., Moine, H., Prats, H. and Prats, A.C.: « Internal ribosome entry site structural motifs conserved among mammalian Fibroblast growth factor 1 alternatively spliced mRNAs » Mol Cell Biol 24 (2004) 7622-7635.

Merten, O.-W.: « State-of-art of the production of retroviral vectors » J Gene Med 6 (2004) S105-24.

Mezzina, M.: « The Second European Conference & Course on pre-clinical gene transfer to train researchers on gene therapy » Gene Therapy 11 (2004) S1-S2.

Millot, G.A., Vainchenker, W., Dumenil, D. and Svinarchuk, F.: « Differential signalling of NH2-terminal flag-labelled thrombopoietin receptor activated by TPO or anti-FLAG antibodies » Cell Signal 16 (2004) 355-63.

Movassagh, M., Laderach, D. and Galy, A.: « Proteins of the Ikaros family control dendritic cell maturation required to induce optimal Th1 T cell differentiation » Int Immunol 16 (2004) 867-75.

Movassagh, M., Spatz, A., Davoust, J., Lebecque, S., Romero, P., Pittet, M., Rimoldi, D., Lienard, D., Gugerli, O., Ferradini, L., Robert, C., Avril, M.F., Zitvogel, L. and Angevin, E.: « Selective accumulation of mature DC-Lamp+ dendritic cells in tumor sites is associated with efficient T-cell-mediated antitumor response and control of metastatic dissemination in melanoma » Cancer Res 64 (2004) 2192-8.

Perez, N., Bigey, P., Scherman, D., Danos, O., Piechaczyk, M. and Pelegrin, M.: « Regulatable systemic production of monoclonal antibodies by in vivo muscle electroporation » Genet Vaccines Ther 2 (2004) 2.

Raslova, H., Komura, E., Le Couédic, J.P., Larbret, F., Debili, N., Feunteun, J., Danos, O., Albagli, O., Vainchenker, W. and Favier, R.: « FLI1 monoallelic expression combined with its hemizygous loss underlies Paris-Trousseau/Jacobsen thrombopenia. » J Clin Invest 114 (2004) 77-84.

Salaun, B., de Saint-Vis, B., Pacheco, N., Pacheco, Y., Riesler, A., Isaac, S., Leroux, C., Clair-Moninot, V., Pin, J.J., Griffith, J., Treilleux, I., Goddard, S., Davoust, J., Kleijmeer, M. and Lebecque, S.: « CD208/dendritic cell-lysosomal associated membrane protein is a marker of normal and transformed type II pneumocytes » Am J Pathol 164 (2004) 861-71.

Taveau, M., Stockholm, D., Marchand, S., Roudaut, C., Le Bert, M. and Richard, I.: « Bidirectional transcriptional activity of the Pgk1 promoter and transmission ratio distortion in Capn3-deficient mice » Genomics 84 (2004) 592-5.

Tazir, M., Azzedine, H., Assami, S., Sindou, P., Nouioua, S., Zemmouri, R., Hamadouche, T., Chaouch, M., Feingold, J., Vallat, J.M., Leguern, E. and Grid, D.: « Phenotypic variability in autosomal recessive axonal Charcot-Marie-Tooth disease due to the R298C mutation in lamin A/C » Brain 127 (2004) 154-63.

Topcu, M., Jobard, F., Halliez, S., Coskun, T., Yalcinkayal, C., Gerceker, F.O., Wanders, R.J., Prud'homme, J.F., Lathrop, M., Ozguc, M. and Fischer, J.: « L-2-Hydroxyglutaric aciduria: identification of a mutant gene C14orf160, localized on chromosome 14q22.1 » Hum Mol Genet 13 (2004) 2803-11.

Vallat, J.M., Grid, D., Magdelaine, C., Sturtz, F. and Tazir, M.: « Autosomal recessive forms of Charcot-Marie-Tooth disease » Curr Neurol Neurosci Rep 4 (2004) 413-9.

2003

Baldeschi, C., Gache, Y., Rattenholl, A., Bouille, P., Danos, O., Ortonne, J.P., Bruckner-Tuderman, L. and Meneguzzi, G.: « Genetic correction of canine dystrophic epidermolysis bullosa mediated by retroviral vectors » Hum Mol Genet 12 (2003) 1897-905.

Brissault, B., Kichler, A., Guis, C., Leborgne, C., Danos, O. and Cheradame, H.: « Synthesis of linear polyethylenimine derivatives for DNA transfection » Bioconjug Chem 14 (2003) 581-7.

Chalouni, C., Banchereau, J., Vogt, A.B., Pascual, V. and Davoust, J.: « Human germinal center B cells differ from naive and memory B cells by their aggregated MHC class II-rich compartments lacking HLA-DO » Int Immunol 15 (2003) 457-66.

Chantret, I., Dancourt, J., Dupre, T., Delenda, C., Bucher, S., Vuillaumier-Barrot, S., Ogier de Baulny, H., Peletan, C., Danos, O., Seta, N., Durand, G., Oriol, R., Codogno, P. and Moore, S.E.: « A deficiency in dolichyl-P-glucose:Glc1Man9GlcNAc2-PP-dolichyl alpha3-glucosyltransferase defines a new subtype of congenital disorders of glycosylation » J Biol Chem 278 (2003) 9962-71.

Chaouch, M., Allal, Y., De Sandre-Giovannoli, A., Vallat, J.M., Amer-el-Khedoud, A., Kassouri, N., Chaouch, A., Sindou, P., Hammadouche, T., Tazir, M., Levy, N. and Grid, D.: « The phenotypic manifestations of autosomal recessive axonal Charcot-Marie-Tooth due to a mutation in Lamin A/C gene » Neuromuscul Disord 13 (2003) 60-7.

Coeytaux, E., Coulaud, D., Le Cam, E., Danos, O. and Kichler, A.: « The cationic amphipathic alpha-helix of HIV-1 Viral protein R (Vpr) binds to nucleic acids, permeabilizes membranes, and efficiently transfects cells » J Biol Chem 278 (2003) 18110-18116.

Combaret, L., Bechet, D., Claustre, A., Taillandier, D., Richard, I. and Attaix, D.: « Down-regulation of genes in the lysosomal and ubiquitin-proteasome proteolytic pathways in calpain-3-deficient muscle » Int J Biochem Cell Biol 35 (2003) 676-84.

De Sandre-Giovannoli, A., Chaouch, M., Boccaccio, I., Bernard, R., Delague, V., Grid, D., Vallat, J.M., Levy, N. and Megarbane, A.: « Phenotypic and genetic exploration of severe demyelinating and secondary axonal neuropathies resulting from GDAP1 nonsense and splicing mutations » J Med Genet 40 (2003) e87.

Douar, A.M., Poulard, K. and Danos, O.: « Deleterious effect of peptide insertions in a permissive site of the AAV2 capsid » Virology 309 (2003) 203-8.

Fougerousse, F., Gonin, P., Durand, M., Richard, I. and Raymackers, J.M.: « Force impairment in calpain 3-deficient mice is not correlated with mechanical disruption » Muscle Nerve 27 (2003) 616-623.

Fruci, D., Lauvau, G., Saveanu, L., Amicosante, M., Butler, R.H., Polack, A., Ginhoux, F., Lemonnier, F., Firat, H. and van Endert, P.M.: « Quantifying recruitment of cytosolic peptides for HLA class I presentation: impact of TAP transport » J Immunol 170 (2003) 2977-84.

Ginhoux, F., Doucet, C., Leboeuf, M., Lemonnier, F.A., Danos, O., Davoust, J. and Firat, H.: « Identification of an HLA-A*0201-restricted epitopic peptide from human dystrophin: application in duchenne muscular dystrophy gene therapy » Mol Ther 8 (2003) 274-83.

Gross, D.A., Leboeuf, M., Gjata, B., Danos, O. and Davoust, J.: « CD4+CD25+ regulatory T cells inhibit immune mediated transgene rejection » Blood 102 (2003) 4326-4328.

Guermonprez, P., Saveanu, L., Kleijmeer, M., Davoust, J., Van Endert, P. and Amigorena, S.: « ER-phagosome fusion defines an MHC class I cross-presentation compartment in dendritic cells » Nature 425 (2003) 397-402.

Jobard, F., Bouadjar, B., Caux, F., Hadj-Rabia, S., Has, C., Matsuda, F., Weissenbach, J., Lathrop, M., Prud'homme, J.F. and Fischer, J.: « Identification of mutations in a new gene encoding a FERM family protein with a pleckstrin homology domain in Kindler syndrome » Hum Mol Genet 12 (2003) 925-35.

Karsenty, E., Barillot, E., Tosser-Klopp, G., Lahbib-Mansais, Y., Milan, D., Hatey, F., Cirera, S., Sawera, M., Jorgensen, C.B., Chowdhary, B., Fredholm, M., Wimmers, K., Ponsuksili, S., Davoli, R., Fontanesi, L., Braglia, S., Zambonelli, P., Bigi, D., Neuenschwander, S. and Gellin, J.L.: « The GENETPIG database: a tool for comparative mapping in pig (Sus scrofa) » Nucleic Acids Research 31 (2003) 138-141.

Kichler, A., Bechinger, B. and Danos, O.: « Des peptides cationiques antibactériens comme vecteurs de transfert de gènes » Med Sci (Paris) 19 (2003) 1046-47.

Kichler, A., Leborgne, C., März, J., Danos, O. and Bechinger, B.: « Histidine-rich amphipathic peptide antibiotics promote efficient delivery of DNA into mammalian cells » Proc Natl Acad Sci USA 100 (2003) 1564-1568.

Kichler, A., Sabourault, N., Decor, R., Leborgne, C., Schmutz, M., Valleix, A., Danos, O., Wagner, A. and Mioskowski, C.: « Preparation and evaluation of a new class of gene transfer reagents: poly(-alkylaminosiloxanes) » J Control Release 93 (2003) 403-14.

Laderach, D., Compagno, D., Danos, O., Vainchenker, W. and Galy, A.: « RNA Interference Shows Critical Requirement for NF-kappaB p50 in the Production of IL-12 by Human Dendritic Cells » J Immunol 171 (2003) 1750-7.

Lefèvre, C., Audebert, S., Jobard, F., Bouadjar, B., Lakhdar, H., Boughdene-Stambouli, O., Blanchet-Bardon, C., Heilig, R., Foglio, M., Weissenbach, J., Lathrop, M., Prud'homme, J.F. and Fischer, J.: « Mutations in the transporter ABCA12 are associated with lamellar ichthyosis type 2 » Hum Mol Genet 12 (2003) 2369-78.

Marchand, S., Hajdari, P., Hackman, P., Udd, B. and Richard, I.: « Touch-down method for high-performance sequencing of polymerase chain reaction products » Anal Biochem 315 (2003) 270-2.

Marrakchi, S., Audebert, S., Bouadjar, B., Has, C., Lefevre, C., Munro, C., Cure, S., Jobard, F., Morlot, S., Hohl, D., Prud'homme, J.F., Zahaf, A., Turki, H. and Fischer, J.: « Novel mutations in the gene encoding secreted lymphocyte antigen-6/urokinase-type plasminogen activator receptor-related protein-1 (SLURP-1) and description of five ancestral haplotypes in patients with Mal de Meleda » J Invest Dermatol 120 (2003) 351-5.

Merten, O.-W.: « Métabolisme cellulaire et besoin nutritif des cellules en culture » In: Barlovatz-Meinon, G. and Adolphe, M. (Eds.), Techniques en Cultures de cellules animales: méthodologies, applications. Les Editions Inserm, Paris, 2003, pp. 99-160.

Nabbout, R., Gennaro, E., Dalla Bernardina, B., Dulac, O., Madia, F., Bertini, E., Capovilla, G., Chiron, C., Cristofori, G., Elia, M., Fontana, E., Gaggero, R., Granata, T., Guerrini, R., Loi, M., La Selva, L., Lispi, M.L., Matricardi, A., Romeo, A., Tzolas, V., Valseriati, D., Veggiotti, P., Vigevano, F., Vallee, L., Dagna Bricarelli, F., Bianchi, A. and Zara, F.: « Spectrum of SCN1A mutations in severe myoclonic epilepsy of infancy » Neurology 60 (2003) 1961-7.

Nabbout, R., Kozlovski, A., Gennaro, E., Bahi-Buisson, N., Zara, F., Chiron, C., Bianchi, A., Brice, A., Leguern, E. and Dulac, O.: « Absence of mutations in major GEFS+ genes in myoclonic astatic epilepsy » Epilepsy Res 56 (2003) 127-33.

Pagès, J.C. and Danos, O.: « Retrovectors go forward » In: Rolland, A. and Sullivan, S.M. (Eds.), Pharmaceutical Gene Delivery Systems. Marcel Dekker, Inc, Basel, 2003, pp. 215-244.

Rohrlich, P.S., Cardinaud, S., Firat, H., Lamari, M., Briand, P., Escriou, N. and Lemonnier, F.A.: « HLA-B*0702 transgenic, H-2K(b)D(b) double-knockout mice: phenotypical and functional characterization in response to influenza virus » Int Immunol 15 (2003) 765-772.

Sander, T., Windemuth, C., Schulz, H., Saar, K., Gennaro, E., Riggio, C., Bianchi, A., Zara, F., Rudolf, G., Picard, F., Bulteau, C., Kaminska, A., Cieuta, C., Prud'homme, J.F., Dulac, O., Bate, L., Robinson, R., Gardiner, R.M., Covanis, A., De Haan, G.J., Janssen, G.A., Van Erp, M.G., Boezeman, E.H., Lindhout, D., Heils, A., Nurnberg, P. and Janz, D.: « Exploration of a putative susceptibility locus for idiopathic generalized epilepsy on chromosome 8p12 » Epilepsia 44 (2003) 32-9.

Sandre-Giovannoli, A.D., Chaouch, M., Boccaccio, I., Bernard, R., Delague, V., Grid, D., Vallat, J.M., Levy, N. and Megarbane, A.: « Phenotypic and genetic exploration of severe demyelinating and secondary axonal neuropathies resulting from GDAP1 nonsense and splicing mutations » Journal Of Medical Genetics 40 (2003).

Scardino, A., Correale, P., Firat, H., Pellegrini, M., Kosmatopoulos, K., Opolon, P., Alves, P., Zurbriggen, R., Gluck, R., Lemonnier, F.A., Francini, G. and Cusi, M.G.: « In vivo study of the GC90/IRIV vaccine for immune response and autoimmunity into a novel humanised transgenic mouse » Br J Cancer 89 (2003) 199-205.

Seifert, U., Maranon, C., Shmueli, A., Desoutter, J.F., Wesoloski, L., Janek, K., Henklein, P., Diescher, S., Andrieu, M., de la Salle, H., Weinschenk, T., Schild, H., Laderach, D., Galy, A., Haas, G., Kloetzel, P.M., Reiss, Y. and Hosmalin, A.: « An essential role for tripeptidyl peptidase in the generation of an MHC class I epitope » Nat Immunol 4 (2003) 375-9.

Stumptner-Cuvelette, P., Jouve, M., Helft, J., Dugast, M., Glouzman, A.S., Jooss, K., Raposo, G. and Benaroch, P.: « Human immunodeficiency virus-1 Nef expression induces intracellular accumulation of multivesicular bodies and major histocompatibility complex class II complexes: potential role of phosphatidylinositol 3-kinase » Mol Biol Cell 14 (2003) 4857-70.

Taveau, M., Bourg, N., Sillon, G., Roudaut, C., Bartoli, M. and Richard, I.: « Calpain 3 is activated through autolysis within the active site and lyses sarcomeric and sarcolemmal components » Mol Cell Biol 23 (2003) 9127-35.

Van Den Bergh, P.Y., Bouquiaux, O., Verellen, C., Marchand, S., Richard, I., Hackman, P. and Udd, B.: « Tibial muscular dystrophy in a Belgian family » Ann Neurol 54 (2003) 248-51.

2002

Bessis, N., Doucet, C., Cottard, V., Douar, A.M., Firat, H., Jorgensen, C., Mezzina, M. and Boissier, M.C.: « Gene therapy for rheumatoid arthritis » J Gene Med 4 (2002) 581-91.

Chantret, I., Dupre, T., Delenda, C., Bucher, S., Dancourt, J., Barnier, A., Charollais, A., Heron, D., Bader-Meunier, B., Danos, O., Seta, N., Durand, G., Oriol, R., Codogno, P. and Moore, S.E.: « Congenital disorders of glycosylation type Ig is defined by a deficiency in dolichyl-P-mannose:Man7GlcNAc2-PP-dolichyl mannosyltransferase » J Biol Chem 277 (2002) 25815-25822.

De Lonlay, P., Mugnier, C., Sanlaville, D., Chantrel-Groussard, K., Benit, P., Lebon, S., Chretien, D., Kadhom, N., Saker, S., Gyapay, G., Romana, S., Weissenbach, J., Munnich, A., Rustin, P. and Rotig, A.: « Cell complementation using Genebridge 4 human:rodent hybrids for physical mapping of novel mitochondrial respiratory chain deficiency genes » Hum Mol Genet 11 (2002) 3273-81.

De Sandre-Giovannoli, A., Chaouch, M., Kozlov, S., Vallat, J.M., Tazir, M., Kassouri, N., Szepetowski, P., Hammadouche, T., Vandenberghe, A., Stewart, C.L., Grid, D. and Levy, N.: « Homozygous defects in LMNA, encoding lamin A/C nuclear-envelope proteins, cause autosomal recessive axonal neuropathy in human (Charcot-Marie-Tooth disorder type 2) and mouse » Am J Hum Genet 70 (2002) 726-736 (Erratum in: Am J Hum Genet 2002 Apr;70(4):1075.

Delenda, C., Audit, M. and Danos, O.: « Biosafety issues in lentivector production » In: Trono, D. (Ed.), Lentiviral Vectors. Springer-Verlag, Berlin, 2002, pp. 123-141.

Duan, M.L., Bordet, T., Mezzina, M., Kahn, A. and Ulfendahl, M.: « Adenoviral and adeno-associated viral vector mediated gene transfer in the guinea pig cochlea » Neuroreport 13 (2002) 1295-1299.

Feasson, L., Stockholm, D., Freyssenet, D., Richard, I., Duguez, S., Beckmann, J.S. and Denis, C.: « Molecular adaptations of neuromuscular disease-associated proteins in response to eccentric exercise in human skeletal muscle » J Physiol 543 (2002) 297-306.

Firat, H., Cochet, M., Rohrlich, P.S., Garcia-Pons, F., Darche, S., Danos, O., Lemonnier, F.A. and Langlade-Demoyen, P.: « Comparative analysis of the CD8(+) T cell repertoires of H-2 class I wild-type/HLA-A2.1 and H-2 class I knockout/HLA-A2.1 transgenic mice » Int Immunol 14 (2002) 925-934.

Firat, H., Zennou, V., Garcia-Pons, F., Ginhoux, F., Cochet, M., Danos, O., Lemonnier, A.F., Langlade-Demoyen, P. and Charneau, P.: « Use of a lentiviral flap vector for induction of CTL immunity against melanoma. Perspectives for immunotherapy » J Gene Med 4 (2002) 38-45.

Fougerousse, F., Durand, M., Lopez, S., Suel, L., Demignon, J., Thornton, C., Ozaki, H., Kawakami, K., Barbet, P., Beckmann, J.S. and Maire, P.: « Six and Eya expression during human somitogenesis and MyoD gene family activation » J Muscle Res Cell Motil 23 (2002) 255-264.

Genin, E., Huebner, A., Jaillard, C., Faure, A., Halaby, G., Saka, N., Clark, A.J.L., Durand, P., Begeot, M. and Naville, D.: « Linkage of one gene for familial glucocorticoid deficiency type 2 (FGD2) to chromosome 8q and further evidence of heterogeneity » Human Genetics 111 (2002) 428-434.

Hackman, P., Vihola, A., Haravuori, H., Marchand, S., Sarparanta, J., De Seze, J., Labeit, S., Witt, C., Peltonen, L., Richard, I. and Udd, B.: « Tibial muscular dystrophy is a titinopathy caused by mutations in TTN, the gene encoding the giant skeletal-muscle protein titin » Am J Hum Genet 71 (2002) 492-500.

Hamel, Y., Blake, N., Gabrielsson, S., Haigh, T., Jooss, K., Martinache, C., Caillat-Zucman, S., Rickinson, A.B., Hacein-Bey, S., Fischer, A. and Cavazzana-Calvo, M.: « Adenovirally transduced dendritic cells induce bispecific cytotoxic T lymphocyte responses against adenovirus and cytomegalovirus pp65 or against adenovirus and Epstein-Barr virus EBNA3C protein: a novel approach for immunotherapy » Hum Gene Ther 13 (2002) 855-866.

Hernandez, J., Garcia-Pons, F., Lone, Y.C., Firat, H., Schmidt, J.D., Langlade-Demoyen, P. and Zanetti, M.: « Identification of a human telomerase reverse transcriptase peptide of low affinity for HLA A2.1 that induces cytotoxic T lymphocytes and mediates lysis of tumor cells » Proc Natl Acad Sci USA 99 (2002) 12275-12280.

Jobard, F., Lefèvre, C., Karaduman, A., Blanchet-Bardon, C., Emre, S., Weissenbach, J., Özgüc, M., Lathrop, M., Prud'homme, J.F. and Fischer, J.: « Lipoxygenase-3 (ALOXE3) and 12(R)-lipoxygenase (ALOX12B) are mutated in non-bullous congenital ichthyosiform erythroderma (NCIE) linked to chromosome 17p13.1 » Hum Mol Genet 11 (2002) 107-113.

Kichler, A., Chillon, M., Leborgne, C., Danos, O. and Frisch, B.: « Intranasal gene delivery with a polyethylenimine-PEG conjugate » J Control Release 81 (2002) 379-388.

Kissa, K., Mordelet, E., Soudais, C., Kremer, E., Demeneix, B., Brulet, P. and Coen, L.: « In vivo neuronal tracing with GFP-TTC gene delivery » Mol Cell Neurosci 20 (2002) 627-637.

Lambolez, F., Jooss, K., Vasseur, F. and Sarukhan, A.: « Tolerance induction to self antigens by peripheral dendritic cells » Eur J Immunol 32 (2002) 2588-2597.

Li Duan, M., Bordet, T., Mezzina, M., Kahn, A. and Ulfendahl, M.: « Adenoviral and adeno-associated viral vector mediated gene transfer in the guinea pig cochlea » Neuroreport 13 (2002) 1295-1299.

Mahe, E., Lahfa, M., Mansouri, S., Mosharraf-Olmolk, H., Le Rebours, J., Prud'homme, J.F. and Fischer, J.: « Physicians' response to a letter to confirm diagnosis in a genetic study of psoriasis » Eur J Dermatol 12 (2002) 66-69.

Martin-Touaux, E., Puech, J.P., Chateau, D., Emiliani, C., Kremer, E.J., Raben, N., Tancini, B., Orlacchio, A., Kahn, A. and Poenaru, L.: « Muscle as a putative producer of acid alpha-glucosidase for glycogenosis type II gene therapy » Hum Mol Genet 11 (2002) 1637-1645.

Mathews, L.C., Gray, J.T., Gallagher, M.R. and Snyder, R.O.: « Recombinant adeno-associated viral vector production using stable packaging and producer cell lines » In: Phillips, I. (Ed.), Gene Therapy Methods. Academic Press, San Diego, 2002, pp. 393-413.

Merten, O.-W.: « Development of serum-free media for cell growth and production of viruses / viral vaccines – safety issues of animal products used in serum-free media » In: Brown, F., Hendriksen, C., Sesardic, D. and Cussler, K. (Eds.), Advancing Science and Elimination of the Use of Laboratory Animals for Development and Control of Vaccines and Hormones – Symposium, Utrecht, November 2001: Proceedings. S. Karger AG, Basel, 2002, pp. 233-257.

Mezzina, M. and Danos, O.: « Five years of vector service for gene therapy » Trends Genet 18 (2002) 118-119.

Mezzina, M.: « First meeting of the Gene Vector Production network, 4-5 October 2001, Evry, France » J Gene Med 4 (2002) 105-108.

Morante-Redolat, J.M., Gorostidi-Pagola, A., Piquer-Sirerol, S., Saenz, A., Poza, J.J., Galan, J., Gesk, S., Sarafidou, T., Mautner, V.F., Binelli, S., Staub, E., Hinzmann, B., French, L., Prud'homme, J.F., Passarelli, D., Scannapieco, P., Tassinari, C.A., Avanzini, G., Marti-Masso, J.F., Kluwe, L., Deloukas, P., Moschonas, N.K., Michelucci, R., Siebert, R., Nobile, C., Perez-Tur, J. and Lopez de Munain, A.: « Mutations in the LGI1/Epitempin gene on 10q24 cause autosomal dominant lateral temporal epilepsy » Hum Mol Genet 11 (2002) 1119-1128.

Moulard, B., Genton, P., Grid, D., Jeanpierre, M., Ouazzani, R., Mrabet, A., Morris, M., LeGuern, E., Dravet, C., Mauguiere, F., Utermann, B., Baldy-Moulinier, M., Belaidi, H., Bertran, F., Biraben, A., Ali Cherif, A., Chkili, T., Crespel, A., Darcel, F., Dulac, O., Geny, C., Humbert-Claude, V., Kassiotis, P., Buresi, C. and Malafosse, A.: « Haplotype study of West European and North African Unverricht-Lundborg chromosomes: evidence for a few founder mutations » Hum Genet 111 (2002) 255-62.

Nabbout, R., Prud'homme, J.F., Herman, A., Feingold, J., Brice, A., Dulac, O. and LeGuern, E.: « A locus for simple pure febrile seizures maps to chromosome 6q22-q24 » Brain 125 (2002) 2668-80.

Peltékian, E., Garcia, L. and Danos, O.: « Neurotropism and retrograde axonal transport of a canine adenoviral vector: A tool for targeting key structures undergoing neurodegenerative processes » Mol Ther 5 (2002) 25-32.

Perez, N., Plence, P., Millet, V., Greuet, D., Minot, C., Noel, D., Danos, O., Jorgensen, C. and Apparailly, F.: « Tetracycline transcriptional silencer tightly controls transgene expression after in vivo intramuscular electrotransfer: application to interleukin 10 therapy in experimental arthritis » Hum Gene Ther 13 (2002) 2161-72.

Robertson, A., Perea, J., Tolmachova, T., Thomas, P.K. and Huxley, C.: « Effects of mouse strain, position of integration and tetracycline analogue on the tetracycline conditional system in transgenic mice » Gene 282 (2002) 65-74.

Sander, T., Windemuth, C., Schulz, H., Saar, K., Gennaro, E., Bianchi, A., Zara, F., Bulteau, C., Kaminska, A., Ville, D., Cieuta, C., Prud'homme, J.F., Dulac, O., Bate, L., Gardiner, R.M., de Haan, G.J., Janssen, G.A., Witte, J., Halley, D.J., Lindhout, D., Wienker, T.F. and Janz, D.: « No evidence for a susceptibility locus for idiopathic generalized epilepsy on chromosome 18q21.1 » Am J Med Genet 114 (2002) 673-8.

Schneider, H., Muhle, C., Douar, A.M., Waddington, S., Jiang, Q.J., Von der Mark, K., Coutelle, C. and Rascher, W.: « Sustained delivery of therapeutic concentrations of human clotting factor IX – a comparison of adenoviral and AAV vectors administered in utero » J Gene Med 4 (2002) 46-53.

Spencer, M.J., Guyon, J.R., Sorimachi, H., Potts, A., Richard, I., Herasse, M., Chamberlain, J., Dalkilic, I., Kunkel, L.M. and Beckmann, J.S.: « Stable expression of calpain 3 from a muscle transgene in vivo: immature muscle in transgenic mice suggests a role for calpain 3 in muscle maturation » Proc Natl Acad Sci USA 99 (2002) 8874-8879.

Taveau, M., Stockholm, D., Spencer, M. and Richard, I.: « Quantification of splice variants using molecular beacon or scorpion primers » Anal Biochem 305 (2002) 227-235.

Tazir, M., Vallat, J.M., Bomont, P., Zemmouri, R., Sindou, P., Assami, S., Nouioua, S., Hammadouche, T., Grid, D. and Koenig, M.: « Genetic heterogeneity in giant axonal neuropathy: an Algerian family not linked to chromosome 16q24.1 » Neuromuscul Disord 12 (2002) 849-52.

Viollet, L., Barois, A., Rebeiz, J.G., Rifai, Z., Burlet, P., Zarhrate, M., Vial, E., Dessainte, M., Estournet, B., Kleinknecht, B., Pearn, J., Adams, R.D., Urtizberea, J.A., Cros, D.P., Bushby, K., Munnich, A. and Lefebvre, S.: « Mapping of autosomal recessive chronic distal spinal muscular atrophy to chromosome 11q13 » Ann Neurol 51 (2002) 585-592.

Wesa, A. and Galy, A.: « Increased production of pro-inflammatory cytokines and enhanced T cell responses after activation of human dendritic cells with IL-1 and CD40 ligand » BMC Immunol 3 (2002) 14.

Windemuth, C., Schulz, H., Saar, K., Gennaro, E., Bianchi, A., Zara, F., Bulteau, C., Kaminska, A., Ville, D., Cieuta, C., Nabbout-Tarantino, R., Prud'homme, J.F., Dulac, O., Bate, L., Gardiner, R., Lindhout, D., Wienker, T., Janz, D. and Sander, T.: « No evidence for a susceptibility locus for idiopathic generalized epilepsy on chromosome 5 in families with typical absence seizures » Epilepsy Res 51 (2002) 23-9.

Yates, F., Malassis-Seris, M., Stockholm, D., Bouneaud, C., Larousserie, F., Noguiez-Hellin, P., Danos, O., Kohn, D.B., Fischer, A., De Villartay, J.P. and Cavazzana-Calvo, M.: « Gene therapy of RAG-2-/-mice: sustained correction of the immunodeficiency » Blood 100 (2002) 3942-3949.

2001

Achard, F., Vaysseix, G. and Barillot, E.: « XML, bioinformatics and data integration » Bioinformatics 17 (2001) 115-125.

Aoki, M., Liu, J., Richard, I., Bashir, R., Britton, S., Keers, S.M., Oeltjen, J., Brown, H.E., Marchand, S., Bourg, N., Beley, C., McKenna-Yasek, D., Arahata, K., Bohlega, S., Cupler, E., Illa, I., Majneh, I., Barohn, R.J., Urtizberea, J.A., Fardeau, M., Amato, A., Angelini, C., Bushby, K., Beckmann, J.S. and Brown, R.H., Jr.: « Genomic organization of the dysferlin gene and novel mutations in Miyoshi myopathy » Neurology 57 (2001) 271-8.

Baghdiguian, S., Richard, I., Martin, M., Coopman, P., Beckmann, J.S., Mangeat, P. and Lefranc, G.: « Pathophysiology of limb girdle muscular dystrophy type 2A: hypothesis and new insights into the IkappaBalpha/NF-kappaB survival pathway in skeletal muscle » J Mol Med 79 (2001) 254-61.

Balsa, A., Barrera, P., Westhovens, R., Alves, H., Maenaut, K., Pascual-Salcedo, D., Cornelis, F., Bardin, T., Riente, L., Radstake, T.R.D.J., De Almeida, G., Lepage, V., Stravopoulos, C., Spaepen, M., Lopes-Vaz, A., Charron, D., Martinez, M., Prud'homme, J.F., Migliorini, P. and Fritz, P.: « Clinical and immunogenetic characteristics of European multicase rheumatoid arthritis families » Ann Rheum Dis 60 (2001) 573-576.

Barrera, P., Balsa, A., Alves, H., Westhovens, R., Maenaut, K., Cornelis, F., Fritz, P., Bardin, T., Ceu Maia, M., Lopes-Vaz, A., Pascual Salcedo, D., de la Concha, E., Radstake, T., van de Putte, L.B., Migliorini, P., Prud'homme, J.F., Charron, D., Spyropoulou, M., Mendes, A., Spaepen, M., Martinez, M. and Stavropoulos, C.: « Noninherited maternal antigens do not increase the susceptibility for familial rheumatoid arthritis. European Consortium on Rheumatoid Arthritis Families (ECRAF) » J Rheumatol 28 (2001) 968-974.

Barrera, P., Faure, S., Prud'homme, J.F., Balsa, A., Migliorini, P., Chimenti, D., Radstake, T.R., van de Putte, L.B., Pascual-Salcedo, D., Westhovens, R., Maenaut, K., Alves, H., Lopes-Vaz, A., Stravopoulos, C., Spyropoulou, M., Fritz, P., Bardin, T., Charron, D., Lepage, V., Alibert, Martinez, M. and Cornelis, F.: « European genetic study on rheumatoid arthritis: is there a linkage of the interleukin-1 (IL-1), IL-10 or IL-4 genes to RA? » Clin Exp Rheumatol 19 (2001) 709-714.

Baulac, S., Huberfeld, G., Gourfinkel-An, I., Mitropoulou, G., Beranger, A., Prud'homme, J.F., Baulac, M., Brice, A., Bruzzone, R. and LeGuern, E.: « First genetic evidence of GABAa receptor dysfunction in epilepsy: A mutation in the gamma2-subunit gene » Nat Genet 28 (2001) 46-48.

Baulac, S., Picard, F., Herman, A., Feingold, J., Genin, E., Hirsch, E., Prud'homme, J.F., Baulac, M., Brice, A. and LeGuern, E.: « Evidence for a digenic inheritance in a family with both febrile convulsions and temporal lobe epilepsy implicating chromosomes 18qter and 1q25-q31 » Ann Neurol 49 (2001) 786-792.

Ben-Arieh, S.V., Zimerman, B., Smorodinsky, N.I., Yaacubovicz, M., Schechter, C., Bacik, I., Gibbs, J., Bennink, J.R., Yewdell, J.W., Coligan, J.E., Firat, H., Lemonnier, F. and Ehrlich, R.: « Human cytomegalovirus protein US2 interferes with the expression of human HFE, a nonclassical class I major histocompatibility complex molecule that regulates iron homeostasis » J Virol 75 (2001) 10557-62.

Brockington, M., Yuva, Y., Prandini, P., Brown, S.C., Torelli, S., Benson, M.A., Herrmann, R., L.V., A., Bashir, R., Burgunder, J.M., Fallet, S., Romero, N., Fardeau, M., Straub, V., Storey, G., Pollitt, C., Richard, I., Sewry, C.A., Bushby, K., Voit, T., Blake, D.J. and Muntoni, F.: « Mutations in the fukutin-related protein gene (FKRP) identify limb girdle muscular dystrophy 2I as a milder allelic variant of congenital muscular dystrophy MDC1C » Hum Mol Genet 10 (2001) 2851-2859.

Chillon, M. and Kremer, E.J.: « Trafficking and propagation of canine adenovirus vectors lacking a known integrin-interacting motif » Hum Gene Ther 12 (2001) 1815-1823.

Chillon, M. and Kremer, E.J.: « Trafficking and propagation of canine adenovirus vectors lacking a known integrin-interacting motif » Human Gene Therapy 12 (2001) 1815-1823.

Coutelle, C., Themis, M., Schneider, H., Kieserud, T., Cook, T., Douar, A.M., Hanson, M., Pavirani, A. and Rodeck, C.: « Fetal somatic gene therapy – A preventive approach to the treatment of genetic disease » In: Holzgreve, W. and Lessl, M. (Eds.), Stem cells from cord blood, in utero stem cell development, and transplantation-inclusive gene therapy. Springer-Verlag, Berlin, 2001, pp. 99-114.

Douar, A.M. and Danos, O.: « Transfert de gène au moyen de vecteurs adéno-associés » In: Cohen-Haguenauer, O. (Ed.), Thérapie Génique. Editions Tec & Doc/Editions Médicales Internationales, Paris, 2001, pp. 145-154.

Douar, A.M., Poulard, K., Stockholm, D. and Danos, O.: « Intracellular trafficking of adeno-associated virus vectors: Routing to the late endosomal compartment and proteasome degradation » J Virol 75 (2001) 1824-1833.

Drittanti, L., Jenny, C., Poulard, K., Samba, A., Manceau, P., Soria, N., Vincent, N., Danos, O. and Vega, M.: « Optimised helper virus-free production of high-quality adeno-associated virus vectors » J Gene Med 3 (2001) 59-71.

Firat, H., Tourdot, S., Ureta-Vidal, A., Scardino, A., Suhrbier, A., Buseyne, F., Riviere, Y., Danos, O., Michel, M.L., Kosmatopoulos, K. and Lemonnier, F.A.: « Design of a polyepitope construct for the induction of HLA-A0201-restricted HIV 1-specific CTL responses using HLA-A*0201 transgenic, H-2 class I KO mice » Eur J Immunol 31 (2001) 3064-3074.

Fischer, J., Bouadjar, B., Heilig, R., Huber, M., Lefevre, C., Jobard, F., Macari, F., Bakija-Konsuo, A., Ait-Belkacem, F., Weissenbach, J., Lathrop, M., Hohl, D. and Prud'homme, J.F.: « Mutations in the gene encoding SLURP-1 in Mal de Meleda » Hum Mol Genet 10 (2001) 875-880.

Fougerousse, F., Edom-Vovard, F., Merkulova, T., Ott, M.O., Durand, M., Butler-Browne, G. and Keller, A.: « The muscle-specific enolase is an early marker of human myogenesis » J Muscle Res Cell Motil 22 (2001) 535-544.

Gücüyener, K., Özgül, K., Paternotte, C., Erdem, H., Prud'homme, J.F., Özgüc, M. and Topaloglu, H.: « Autosomal recessive spastic ataxia of Charlevoix-Saguenay in two unrelated Turkish families » Neuropediatrics 32 (2001) 142-146.

Haravuori, H., Vihola, A., Straub, V., Auranen, M., Richard, I., Marchand, S., Voit, T., Labeit, S., Somer, H., Peltonen, L., Beckmann, J.S. and Udd, B.: « Secondary calpain 3 deficiency in 2q-linked muscular dystrophy: Titin is the candidate gene » Neurology 56 (2001) 869-877.

Jooss, K., Gjata, B., Danos, O., Von Boehmer, H. and Sarukhan, A.: « Regulatory function of in vivo anergized CD4+ T cells » Proc Natl Acad Sci USA 98 (2001) 8738-8743.

Kichler, A., Leborgne, C., Coeytaux, E. and Danos, O.: « Polyethylenimine-mediated gene delivery: a mechanistic study » J Gene Med 3 (2001) 135-144.

Lefèvre, C., Blanchet-Bardon, C., Jobard, F., Bouadjar, B., Stalder, J.F., Cure, S., Hoffmann, A., Prud'homme, J.F. and Fischer, J.: « Novel point mutations, deletions, and polymorphisms in the cathepsin C gene in nine families from Europe and North Africa with Papillon-Lefèvre syndrome » J Invest Dermatol 117 (2001) 1657-1661.

Lefèvre, C., Jobard, F., Caux, F., Bouadjar, B., Karaduman, A., Heilig, R., Lakhdar, H., Wollenberg, A., Verret, J.L., Weissenbach, J., Özgüc, M., Lathrop, M., Prud'homme, J.F. and Fischer, J.: « Mutations in CGI-58, the gene encoding a new protein of the esterase/Lipase/Thioesterase subfamily, in Chanarin-Dorfman syndrome » Am J Hum Genet 69 (2001) 1002-1012.

Martinez-Arca, S., Coco, S., Mainguy, G., Schenk, U., Alberts, P., Bouille, P., Mezzina, M., Prochiantz, A., Matteoli, M., Louvard, D. and Galli, T.: « A common exocytotic mechanism mediates axonal and dendritic outgrowth » J Neurosci 21 (2001) 3830-3838.

Merten, O.-W., Cruz, P.E., Rochette, C., Geny-Fiamma, C., Bouquet, C., Gonçalves, D., Danos, O. and Carrondo, M.J.T.: « Comparison of different bioreactor systems for the production of high titer retroviral vectors » Biotechnol Prog 17 (2001) 326-335.

Merten, O.-W., Landric, L. and Danos, O.: « Influence of the metabolic state of packaging cells on retroviral vector production » In: Merten, O.W., Mattanovich, D., Lang, C., Larsson, G., Neubauer, P., Porro, D., Postma, P., Teixeira de Mattos, J. and Cole, J.A. (Eds.), Recombinant protein production with prokaryotic and eukaryotic cells: a comparative view on host physiology. Kluwer Academic Press, Dordrecht, 2001, pp. 301-316.

Mologni, L., Salmikangas, P., Fougerousse, F., Beckmann, J.S. and Carpén, O.: « Developmental expression of myotilin, a gene mutated in limb-girdle muscular distrophy type 1A » Mech Dev 103 (2001) 121-125.

Moore, T., Hecquet, S., McLellann, A., Ville, D., Grid, D., Picard, F., Moulard, B., Asherson, P., Makoff, A.J., McCormick, D., Nashef, L., Froguel, P., Arzimanoglou, A., LeGuern, E. and Bailleul, B.: « Polymorphism analysis of JRK/JH8, the human homologue of mouse jerky, and description of a rare mutation in a case of CAE evolving to JME » Epilepsy Res 46 (2001) 157-167.

Pizzato, M., Merten, O.-W., Blair, E.D. and Takeuchi, Y.: « Development of a suspension packaging cell line for production of high titre, serum-resistant murine leukemia virus vectors » Gene Ther 8 (2001) 737-745.

Richard, I. and Beckmann, J.S.: « Molecular diagnosis of calpainopathies: methods used for detection of mutations in the CAPN3 gene implicated in limb-girdle muscular dystrophy type 2A » In: Anderson, L.V.B. and Bushby, K.M.D. (Eds.), Muscular dystrophy: Methods and protocols. Humana Press, Totowa, USA, 2001, pp. 273-288.

Samakoglu, S., Fattori, E., Lamartina, S., Toniatti, C., Stockholm, D., Heard, J.M. and Bohl, D.: « BetaMinor-globin messenger RNA accumulation in reticulocytes governs improved erythropoiesis in beta-thalassemic mice after erythropoietin complementary DNA electrotransfer in muscles » Blood 97 (2001) 2213-2220.

Sarukhan, A., Camugli, S., Gjata, B., Von Boehmer, H., Danos, O. and Jooss, K.: « Successful interference with cellular immune responses to immunogenic proteins encoded by recombinant viral vectors » J Virol 75 (2001) 269-277.

Sarukhan, A., Soudais, C., Danos, O. and Jooss, K.: « Factors influencing cross-presentation of non-self antigens expressed from recombinant adeno-associated virus vectors » J Gene Med 3 (2001) 260-270.

Scardino, A., Alves, P., Gross, D.A., Tourdot, S., Graff-Dubois, S., Angevin, E., Firat, H., Chouaib, S., Lemonnier, F., Nadler, L.M., Cardoso, A.A. and Kosmatopoulos, K.: « Identification of HER-2/neu immunogenic epitopes presented by renal cell carcinoma and other human epithelial tumors » Eur J Immunol 31 (2001) 3261-3270.

Soudais, C., Boutin, S. and Kremer, E.J.: « Characterization of cis-acting sequences involved in canine adenovirus packaging » Mol Ther 3 (2001) 631-640.

Soudais, C., Laplace-Builhe, C., Kissa, K. and Kremer, E.J.: « Preferential transduction of neurons by canine adenovirus vectors and their efficient retrograde transport in vivo » FASEB J 15 (2001) 2283-2285.

Spirito, F., Meneguzzi, G., Danos, O. and Mezzina, M.: « Cutaneous gene transfer and therapy: the present and the future » J Gene Med 3 (2001) 21-31.

Stockholm D, Herasse M, Marchand S, Praud C, Roudaut C, Richard I, Sebille A, Beckmann JS., « Calpain 3 mRNA expression in mice after denervation and during muscle regeneration » , Am J Physiol Cell Physiol. 2001.

Sylvius, N., Tesson, F., Gayet, C., Charron, P., Benaiche, A., Mangin, L., Peuchmaurd, M., Duboscq-Bidot, L., Feingold, J., Beckmann, J.S., Bouchier, C. and Komajda, M.: « A new locus for autosomal dominant dilated cardiomyopathy identified on chromosome 6q12-q14 » American Journal Of Human Genetics 68 (2001) 241-246.

Talim, B., Ognibene, A., Mattioli, E., Richard, I., Anderson, L.V. and Merlini, L.: « Normal calpain expression in genetically confirmed limb-girdle muscular dystrophy type 2A » Neurology 56 (2001) 692-3.

Tiger, C.F., Fougerousse, F., Grundstrom, G., Velling, T. and Gullberg, D.: « Alpha11beta1 integrin is a receptor for interstitial collagens involved in cell migration and collagen reorganization on mesenchymal nonmuscle cells » Dev Biol 237 (2001) 116-129.

Tores, F. and Barillot, E.: « The art of pedigree drawing: algorithmic aspects » Bioinformatics 17 (2001) 174-179.

Carotti M., et al. (2020) « Combined Use of CFTR Correctors in LGMD2D Myotubes Improves Sarcoglycan Complex Recovery » International journal of molecular sciences 21.