Correction of Multiple Striated Muscles in Murine Pompe Disease Through Adeno-associated Virus-Mediated Gene Therapy

Glycogen storage disease type II (GSD-II; Pompe disease; MIM 232300) stems from the deficiency of acid-α-glucosidase (GAA; acid maltase; EC 3.2.1.20), which primarily involves cardiac and skeletal muscles. We hypothesized that systemic administration of an adeno-associated virus (AAV) vector contain...

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Detalhes bibliográficos
Main Authors: Sun, Baodong, Young, Sarah P., Li, Ping, Di, Chunhui, Brown, Talmage, Salva, Maia Z., Li, Songtao, Bird, Andrew, Yan, Zhen, Auten, Richard, Hauschka, Stephen D., Koeberl, Dwight D.
Formato: Artigo
Idioma:Inglês
Publicado em: 2008
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC2670546/
https://ncbi.nlm.nih.gov/pubmed/18560415
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2008.133
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