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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Main Authors: Bortolanza, Sergia, Nonis, Alessandro, Sanvito, Francesca, Maciotta, Simona, Sitia, Giovanni, Wei, Jessica, Torrente, Yvan, Di Serio, Clelia, Chamberlain, Joel R, Gabellini, Davide
פורמט: Artigo
שפה:Inglês
יצא לאור: Nature Publishing Group 2011
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Original Article
גישה מקוונת:https://ncbi.nlm.nih.gov/pmc/articles/PMC3222524/
https://ncbi.nlm.nih.gov/pubmed/21829175
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2011.153
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