AAV6-mediated Systemic shRNA Delivery Reverses Disease in a Mouse Model of Facioscapulohumeral Muscular Dystrophy

Treatment of dominantly inherited muscle disorders remains a difficult task considering the need to eliminate the pathogenic gene product in a body-wide fashion. We show here that it is possible to reverse dominant muscle disease in a mouse model of facioscapulohumeral muscular dystrophy (FSHD). FSH...

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Detalles Bibliográficos
Autores principales: Bortolanza, Sergia, Nonis, Alessandro, Sanvito, Francesca, Maciotta, Simona, Sitia, Giovanni, Wei, Jessica, Torrente, Yvan, Di Serio, Clelia, Chamberlain, Joel R, Gabellini, Davide
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2011
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3222524/
https://www.ncbi.nlm.nih.gov/pubmed/21829175
http://dx.doi.org/10.1038/mt.2011.153

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