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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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| Main Authors: | , , , , , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Mary Ann Liebert, Inc.
2013
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| Assuntos: | |
| Acesso em linha: | 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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