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Full Functional Rescue of a Complete Muscle (TA) in Dystrophic Hamsters by Adeno-Associated Virus Vector-Directed Gene Therapy

Limb girdle muscular dystrophy (LGMD) 2F is caused by mutations in the δ-sarcoglycan (SG) gene. Previously, we have shown successful application of a recombinant adeno-associated virus (AAV) vector for genetic and biochemical rescue in the Bio14.6 hamster, a homologous animal model for LGMD 2F (J. L...

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Autores principales: Xiao, Xiao, Li, Juan, Tsao, Yeou-Ping, Dressman, Devin, Hoffman, Eric P., Watchko, Jon F.
Formato: Artigo
Lenguaje:Inglês
Publicado: American Society for Microbiology 2000
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Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC111478/
https://ncbi.nlm.nih.gov/pubmed/10627554
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