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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Hlavní autoři: Sun, Baodong, Li, Songtao, Bird, Andrew, Koeberl, Dwight D.
Médium: Artigo
Jazyk:Inglês
Vydáno: 2010
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On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC2988075/
https://ncbi.nlm.nih.gov/pubmed/20686508
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2010.109
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