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Hydrostatic Isolated Limb Perfusion with Adeno-associated Virus Vectors Enhances Correction of Skeletal Muscle in Pompe Disease

Glycogen storage disease type II (GSD-II; Pompe disease; MIM 232300) stems from the inherited deficiency of acid-α-glucosidase (GAA; acid maltase; EC 3.2.1.20), which primarily involves cardiac and skeletal muscles. We hypothesized that hydrostatic isolated limb perfusion (ILP) administration of an...

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Detalles Bibliográficos
Autores principales:	Sun, Baodong, Li, Songtao, Bird, Andrew, Koeberl, Dwight D.
Formato:	Texto
Lenguaje:	English
Publicado:	2010
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2988075/ https://www.ncbi.nlm.nih.gov/pubmed/20686508 http://dx.doi.org/10.1038/gt.2010.109

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