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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.
Béroud C, Tuffery-Giraud S, Matsuo M, Hamroun D, Humbertclaude V, Monnier N, Moizard MP, Voelckel MA, Calemard LM, Boisseau P, Blayau M, Philippe C, Cossée M, Pagès M, Rivier F, Danos O, Garcia L, Claustres M. Béroud C, et al. Among authors: garcia l. Hum Mutat. 2007 Feb;28(2):196-202. doi: 10.1002/humu.20428. Hum Mutat. 2007. PMID: 17041910
Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping.
Goyenvalle A, Vulin A, Fougerousse F, Leturcq F, Kaplan JC, Garcia L, Danos O. Goyenvalle A, et al. Among authors: garcia l. Science. 2004 Dec 3;306(5702):1796-9. doi: 10.1126/science.1104297. Epub 2004 Nov 4. Science. 2004. PMID: 15528407
Immortalized skin fibroblasts expressing conditional MyoD as a renewable and reliable source of converted human muscle cells to assess therapeutic strategies for muscular dystrophies: validation of an exon-skipping approach to restore dystrophin in Duchenne muscular dystrophy cells.
Chaouch S, Mouly V, Goyenvalle A, Vulin A, Mamchaoui K, Negroni E, Di Santo J, Butler-Browne G, Torrente Y, Garcia L, Furling D. Chaouch S, et al. Among authors: garcia l. Hum Gene Ther. 2009 Jul;20(7):784-90. doi: 10.1089/hum.2008.163. Hum Gene Ther. 2009. PMID: 19358679
3,258 results