Publications scientifiques 2018

•  1. 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.
•  2. 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.
•  3. 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.
•  4. 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.
•  5. Colella, P., et al. (2018). « Emerging Issues in AAV-Mediated In Vivo Gene Therapy. » Mol Ther Methods Clin Dev 8: 87-104.
•  6. 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.
•  7. 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.
•  8. 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.
•  9. 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.
• 10. Dorange, F. and C. Le Bec (2018). « Analytical approaches to characterize AAV vector production & purification: Advances and challenges. » Cell & Gene Therapy Insights Latest development in viral & non-viral vector manufacturing: 119-129.
• 11. 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.
• 12. Galibert, L., et al. (2018). « Origins of truncated supplementary capsid proteins in rAAV8 vectors produced with the baculovirus system. » PLoS One 13(11): e0207414.
• 13. Henriques, S. F., et al. (2018). « Different outcome of sarcoglycan missense mutation between human and mouse. » PLoS One 13(1): e0191274.
• 14. 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.
• 15. 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.
• 16. 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.
• 17. 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.
• 18. Kuranda, K., et al. (2018). « Exposure to wild-type AAV drives distinct capsid immunity profiles in humans. » J Clin Invest 128(12): 5267-5279.
• 19.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.
• 20. 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.
• 21. 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.
• 22. Mingozzi, F. (2018). « AAV Immunogenicity: A Matter of Sensitivity. » Mol Ther 26(10): 2335-2336.
• 23. Oates, E. C., et al. (2018). « Congenital Titinopathy: Comprehensive characterization and pathogenic insights. » Ann Neurol 83(6): 1105-1124.
• 24. Paldi, A. (2018). « Conceptual Challenges of the Systemic Approach in Understanding Cell Differentiation. » Methods Mol Biol, Springer. 1702: 27-39.
• 25. 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.
• 26. 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.
• 27. Poletti, V. and F. Mavilio (2018). « Interactions between Retroviruses and the Host Cell Genome. » Mol Ther Methods Clin Dev 8: 31-41.
• 28. 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.
• 29. Ronzitti, G. and F. Mingozzi (2018). « Combination Therapy Is the New Gene Therapy? » Mol Ther 26(1): 12-14.
• 30. Rouillon, J., et al. (2018). « High urinary ferritin reflects myoglobin iron evacuation in DMD patients. » Neuromuscul Disord 28(7): 564-571.
• 31. Toueille, M., et al. (2018). « Development of purification steps for several AAV serotypes using POROS™ CaptureSelect™ AAVX affinity chromatography. » Cell & Gene Therapy Insights 4 (7): 637-645.
• 32. Urbinati, F., et al. (2018). « Gene Therapy for Sickle Cell Disease: A Lentiviral Vector Comparison Study. » Hum Gene Ther 29(10): 1153-1166.
• 33. 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.
• 34. 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.
• 35. 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.
• 36. 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.
• 37. Wightman, R. M., et al. (2018). « How intravesicular composition affects exocytosis. » Pflugers Arch – Eur J Physiol 470: 135-141.