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Hepatorenal Correction in Murine Glycogen Storage Disease Type I With a Double-stranded Adeno-associated Virus Vector

Glycogen storage disease type Ia (GSD-Ia) is caused by the deficiency of glucose-6-phosphatase (G6Pase). Long-term complications of GSD-Ia include life-threatening hypoglycemia and proteinuria progressing to renal failure. A double-stranded (ds) adeno-associated virus serotype 2 (AAV2) vector encodi...

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Detalhes bibliográficos
Main Authors: Luo, Xiaoyan, Hall, Gentzon, Li, Songtao, Bird, Andrew, Lavin, Peter J, Winn, Michelle P, Kemper, Alex R, Brown, Talmage T, Koeberl, Dwight D
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2011
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3222521/
https://ncbi.nlm.nih.gov/pubmed/21730973
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2011.126
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