Forelimb Treatment in a Large Cohort of Dystrophic Dogs Supports Delivery of a Recombinant AAV for Exon Skipping in Duchenne Patients

Duchenne muscular dystrophy (DMD) is a severe muscle-wasting disorder caused by mutations in the dystrophin gene, without curative treatment yet available. Our study provides, for the first time, the overall safety profile and therapeutic dose of a recombinant adeno-associated virus vector, serotype...

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Pubblicato in:Mol Ther
Autori principali: Le Guiner, Caroline, Montus, Marie, Servais, Laurent, Cherel, Yan, Francois, Virginie, Thibaud, Jean-Laurent, Wary, Claire, Matot, Béatrice, Larcher, Thibaut, Guigand, Lydie, Dutilleul, Maeva, Domenger, Claire, Allais, Marine, Beuvin, Maud, Moraux, Amélie, Le Duff, Johanne, Devaux, Marie, Jaulin, Nicolas, Guilbaud, Mickaël, Latournerie, Virginie, Veron, Philippe, Boutin, Sylvie, Leborgne, Christian, Desgue, Diana, Deschamps, Jack-Yves, Moullec, Sophie, Fromes, Yves, Vulin, Adeline, Smith, Richard H, Laroudie, Nicolas, Barnay-Toutain, Frédéric, Rivière, Christel, Bucher, Stéphanie, Le, Thanh-Hoa, Delaunay, Nicolas, Gasmi, Mehdi, Kotin, Robert M, Bonne, Gisèle, Adjali, Oumeya, Masurier, Carole, Hogrel, Jean-Yves, Carlier, Pierre, Moullier, Philippe, Voit, Thomas
Natura: Artigo
Lingua:Inglês
Pubblicazione: Nature Publishing Group 2014
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Original Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC4429735/
https://ncbi.nlm.nih.gov/pubmed/25200009
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2014.151
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