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Comparative impact of AAV and enzyme replacement therapy on respiratory and cardiac function in adult Pompe mice

Pompe disease is an autosomal recessive genetic disorder characterized by a deficiency of the enzyme responsible for degradation of lysosomal glycogen (acid α-glucosidase (GAA)). Cardiac dysfunction and respiratory muscle weakness are primary features of this disorder. To attenuate the progressive a...

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Detalles Bibliográficos
Autores principales:	Falk, Darin J, Soustek, Meghan S, Todd, Adrian Gary, Mah, Cathryn S, Cloutier, Denise A, Kelley, Jeffry S, Clement, Nathalie, Fuller, David D, Byrne, Barry J
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group 2015
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4445006/ https://www.ncbi.nlm.nih.gov/pubmed/26029718 http://dx.doi.org/10.1038/mtm.2015.7

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