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Intrapleural Administration of AAV9 Improves Neural and Cardiorespiratory Function in Pompe Disease

Pompe disease is a neuromuscular disease resulting from deficiency in acid α-glucosidase (GAA), results in cardiac, skeletal muscle, and central nervous system (CNS) pathology. Enzyme replacement therapy (ERT) has been shown to partially correct cardiac and skeletal muscle dysfunction. However, ERT...

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Detalhes bibliográficos
Main Authors: Falk, Darin J, Mah, Cathryn S, Soustek, Meghan S, Lee, Kun-Ze, ElMallah, Mai K, Cloutier, Denise A, Fuller, David D, Byrne, Barry J
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2013
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3776643/
https://ncbi.nlm.nih.gov/pubmed/23732990
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2013.96
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