Enhanced Efficacy of an AAV Vector Encoding Chimeric, Highly-Secreted Acid α-glucosidase in Glycogen Storage Disease Type II

Glycogen storage disease type II (GSD-II; Pompe disease; MIM 232300) is an inherited muscular dystrophy caused by deficiency in the activity of the lysosomal enzyme acid α-glucosidase (GAA). We hypothesized that chimeric GAA containing an alternative signal peptide could increase the secretion of GA...

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Main Authors: Sun, Baodong, Zhang, Haoyue, Benjamin, Daniel K., Brown, Talmage, Bird, Andrew, Young, Sarah P., McVie-Wylie, Alison, Chen, Y-T, Koeberl, Dwight D.
Format: Artigo
Sprog:Inglês
Udgivet: 2006
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC2709843/
https://ncbi.nlm.nih.gov/pubmed/16987711
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2006.08.001
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