Muscle Function Recovery in Golden Retriever Muscular Dystrophy After AAV1-U7 Exon Skipping

Duchenne muscular dystrophy (DMD) is an X-linked recessive disorder resulting from lesions of the gene encoding dystrophin. These usually consist of large genomic deletions, the extents of which are not correlated with the severity of the phenotype. Out-of-frame deletions give rise to dystrophin def...

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Autori principali: Vulin, Adeline, Barthélémy, Inès, Goyenvalle, Aurélie, Thibaud, Jean-Laurent, Beley, Cyriaque, Griffith, Graziella, Benchaouir, Rachid, le Hir, Maëva, Unterfinger, Yves, Lorain, Stéphanie, Dreyfus, Patrick, Voit, Thomas, Carlier, Pierre, Blot, Stéphane, Garcia, Luis
Natura: Artigo
Lingua:Inglês
Pubblicazione: Nature Publishing Group 2012
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Original Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC3498802/
https://ncbi.nlm.nih.gov/pubmed/22968479
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2012.181
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