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Adeno-Associated Virus–Mediated Gene Therapy for Metabolic Myopathy

Metabolic myopathies are a diverse group of rare diseases in which impaired breakdown of stored energy leads to profound muscle dysfunction ranging from exercise intolerance to severe muscle wasting. Metabolic myopathies are largely caused by functional deficiency of a single gene and are generally...

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Detaylı Bibliyografya
Asıl Yazarlar: Mah, Cathryn S., Soustek, Meghan S., Todd, A. Gary, McCall, Angela, Smith, Barbara K., Corti, Manuela, Falk, Darin J., Byrne, Barry J.
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: Mary Ann Liebert, Inc. 2013
Konular:
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC3814817/
https://ncbi.nlm.nih.gov/pubmed/24164240
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2013.2514
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