Impaired clearance of accumulated lysosomal glycogen in advanced Pompe disease despite high-level vector-mediated transgene expression

BACKGROUND: Infantile-onset glycogen storage disease type II (GSD-II; Pompe disease; MIM 232300) causes death early in childhood from cardiorespiratory failure in absence of effective treatment, whereas late-onset Pompe disease causes a progressive skeletal myopathy. The limitations of enzyme replac...

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Hlavní autoři: Sun, Baodong, Zhang, Haoyue, Bird, Andrew, Li, Songtao, Young, Sarah P., Koeberl, Dwight D.
Médium: Artigo
Jazyk:Inglês
Vydáno: 2009
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On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC3622249/
https://ncbi.nlm.nih.gov/pubmed/19621331
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/jgm.1372
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