Bibliography
- Guthleben D, Le Faou O. Une course pour la vie, L’AFM et la recherche biologique et médicale. Armand Colin; Paris: 2011
- Monaco AP, Neve RL, Colletti-Feener C, et al. Isolation of candidate cDNAs for portions of the Duchenne muscular dystrophy gene. Nature 1986;323(6089):646-50
- Chumakov I, Rigault P, Guillou S, et al. Continuum of overlapping clones spanning the entire human chromosome 21q. Nature 1992;359(6394):380-7
- Bellanné-Chantelot C, Lacroix B, Ougen P, et al. Mapping the whole human genome by fingerprinting yeast artificial chromosomes. Cell 1992;70(6):1059-68
- Cohen D, Chumakov I, Weissenbach J. A first-generation physical map of the human genome. Nature 1993;366(6456):698-701
- Weissenbach J, Gyapay G, Dib C, et al. A second-generation linkage map of the human genome. Nature 1992;359(6398):794-801
- Gyapay G, Morissette J, Vignal A, et al. The 1993-94 Généthon human genetic linkage map. Nat Genet 1994;7(2 Spec No):246-339
- Dib C, Fauré S, Fizames C, et al. A comprehensive genetic map of the human genome based on 5,264 microsatellites. Nature 1996;380(6570):152-4
- Goyenvalle A, Vulin A, Fougerousse F, et al. Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping. Science 2004;306(5702):1796-9
- Childers MK, Joubert R, Poulard K, et al. Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy. Sci Transl Med 2014;6(220):220ra10
- Richard I, Broux O, Allamand V, et al. Mutations in the proteolytic enzyme calpain 3 cause limb-girdle muscular dystrophy type 2A. Cell 1995;81(1):27-40
- Fougerousse F, Bartoli M, Poupiot J, et al. Phenotypic correction of α-sarcoglycan deficiency by intra-arterial injection of a muscle specific AAV1 serotype vector. Mol Ther 2007;15(1):53-61
- Roudaut C, Le Roy F, Suel L, et al. Restriction of calpain3 expression to the skeletal muscle prevents cardiac toxicity and corrects pathology in a murine model of limb-girdle muscular dystrophy. Circulation 2013;128(10):1094-104
- Herson S, Hentati F, Rigolet A, et al. A phase I trial of adeno-associated virus serotype 1-γ-sarcoglycan gene therapy for limb girdle muscular dystrophy type 2C. Brain 2012;135(Pt 2):483-92
- Charrier S, Stockholm D, Seye K, et al. A lentiviral vector encoding the human Wiskott-Aldrich syndrome protein corrects immune and cytoskeletal defects in WASP knockout mice. Gene Ther 2005;12(7):597-606
- Galy A, Thrasher AJ. Gene therapy for the Wiskott-Aldrich syndrome. Curr Opin Allergy Clin Immunol 2011;11(6):545-50
- Moors EH, Cohen AF, Schellekens H. Towards a sustainable system of drug development. Drug Discov Today 2014. [Epub ahead of print]
- Kaufmann KB, Büning H, Galy A, et al. Gene therapy on the move. EMBO Mol Med 2013;5(11):1642-61