Scientific publications 2014

  1. 1. Allenbach, Y., W. Chaara, et al. (2014). “Th1 response and systemic treg deficiency in inclusion body myositis.” PLoS One 9(3): e88788.
  2. 2. Basner-Tschakarjan, E. and F. Mingozzi (2014). “Cell-Mediated Immunity to AAV Vectors, Evolving Concepts and Potential Solutions.” Front Immunol 5: 350.
  3. 3. Brayet, J., F. Zehraoui, et al. (2014). “Towards a piRNA prediction using multiple kernel fusion and support vector machine.” Bioinformatics 30(17): i364-i370.
  4. 4. 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.
  5. 5. 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.
  6. 6. 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.” PLoS One 9(7): e101644.
  7. 7. Clermont, D., S. Santoni, et al. (2014). “Assessment of DNA encapsulation, a new room-temperature DNA storage method.” Biopreserv Biobank 12(3): 176-183.
  8. 8. Corre, G., D. Stockholm, et al. (2014). “Stochastic fluctuations and distributed control of gene expression impact cellular memory.” PLoS One 9(12): e115574.
  9. 9. Corre, G., D. Stockholm, et al. (2014). “Variabilité non-génétique dans les cultures cellulaires”. Culture de cellules animales, 3ème édition. G. Barlovatz-Meimon and X. Ronot, Lavoisier S.A.S. / Inserm.
  10. 10. 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.
  11. 11. Evila, A., A. Vihola, et al. (2014). “Atypical phenotypes in titinopathies explained by second titin mutations.” Ann Neurol 75(2): 230-240.
  12. 12. 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.
  13. 13. 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.
  14. 14. 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.
  15. 15. 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.
  16. 16. 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.
  17. 17. 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.
  18. 18. 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.
  19. 19. 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.
  20. 20. Mavilio, F. (2014). “Repairing without cutting: a safer alternative to gene correction?” Mol Ther 22(4): 690-691.
  21. 21. Merten, O. W., W. A. M. Bakker, et al. (2014). “Virus production under suspension conditions”. Industrial scale suspension culture of living cells. H. P. Meyer and D. Schmidhalter, Wiley-VCH Vrlag GmbH & Co. KGaA: 503-554.
  • 22. Merten, O. W., M. Schweizer, et al. (2014). Production and purification of viral vectors and safety considerations related to their use. Advanced textbook on gene transfer, gene therapy and genetic pharmacology. D. Scherman, Imperial College Press, London/U.K.: 493-515.
  • 23. Merten, O. W., M. Schweizer, et al. (2014). “Manufacturing of viral vectors for gene therapy: part I. Upstream processing.” Pharma. Bioprocess. 2(2): 183-203.
  • 24. Merten, O. W., M. Schweizer, et al. (2014). “Manufacturing of viral vectors: part II. Downstream processing and safety aspects.” Pharma. Bioprocess. 2(3): 237-251.
  1. 25. 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.
  2. 26. 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.
  3. 27. 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.
  4. 28. Nathwani, A. C., U. M. Reiss, et al. (2014). “Long-term safety and efficacy of factor IX gene therapy in hemophilia B.” N Engl J Med 371(21): 1994-2004.
  5. 29. Neildez-Nguyen, T. M., J. Bigot, et al. (2014). “Hypoxic culture conditions enhance the generation of regulatory T cells.” Immunology.
  6. 30. Relizani, K., E. Mouisel, et al. (2014). “Blockade of ActRIIB signaling triggers muscle fatigability and metabolic myopathy.” Mol Ther.
  7. 31. Revah, F. (2014). “Genethon: patient-empowered research.” Informa Healthcare: 1-6.
  8. 32. 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.
  9. 33. Rouillon, J., A. Zocevic, et al. (2014). “Proteomics profiling of urine reveals specific titin fragments as biomarkers of Duchenne muscular dystrophy.” Neuromuscul Disord.
  10. 34. 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.
  11. 35. Turchiano, G., M. C. Latella, et al. (2014). “Genomic analysis of sleeping beauty transposon integration in human somatic cells.” PLoS One 9(11): e112712.