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AAV.Dysferlin Overlap Vectors Restore Function in Dysferlinopathy Animal Models

OBJECTIVE: Dysferlinopathies are a family of untreatable muscle disorders caused by mutations in the dysferlin gene. Lack of dysferlin protein results in progressive dystrophy with chronic muscle fiber loss, inflammation, fat replacement, and fibrosis; leading to deteriorating muscle weakness. The o...

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Detalles Bibliográficos
Autores principales:	Sondergaard, Patricia C, Griffin, Danielle A, Pozsgai, Eric R, Johnson, Ryan W, Grose, William E, Heller, Kristin N, Shontz, Kim M, Montgomery, Chrystal L, Liu, Joseph, Clark, Kelly Reed, Sahenk, Zarife, Mendell, Jerry R, Rodino-Klapac, Louise R
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BlackWell Publishing Ltd 2015
Materias:	Research Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4369275/ https://www.ncbi.nlm.nih.gov/pubmed/25815352 http://dx.doi.org/10.1002/acn3.172

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