Long-term effects of systemic gene therapy in a canine model of myotubular myopathy

INTRODUCTION: X-linked myotubular myopathy (XLMTM), a devastating pediatric disease caused by the absence of the protein myotubularin, results from mutations in the MTM1 gene. While there is no cure for XLMTM, we previously reported effects of MTM1 gene therapy using adeno-associated viral (AAV) vec...

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Publicado no:Muscle Nerve
Main Authors: Elverman, Matthew, Goddard, Melissa A, Mack, David, Snyder, Jessica M, Lawlor, Michael W, Meng, Hui, Beggs, Alan H, Buj-Bello, Ana, Poulard, Karine, Marsh, Anthony P, Grange, Robert W, Kelly, Valerie E., Childers, Martin K
Formato: Artigo
Idioma:Inglês
Publicado em: 2017
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5620115/
https://ncbi.nlm.nih.gov/pubmed/28370029
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/mus.25658
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