Complete Normalization of Hepatic G6PC Deficiency in Murine Glycogen Storage Disease Type Ia Using Gene Therapy

Glycogen storage disease type Ia (GSD-Ia) patients deficient in glucose-6-phosphatase-α (G6Pase-α or G6PC) manifest disturbed glucose homeostasis. We examined the efficacy of liver G6Pase-α delivery mediated by AAV-GPE, an adeno-associated virus (AAV) serotype 8 vector expressing human G6Pase-α dire...

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Autori principali: Yiu, Wai Han, Lee, Young Mok, Peng, Wen-Tao, Pan, Chi-Jiunn, Mead, Paul A, Mansfield, Brian C, Chou, Janice Y
Natura: Artigo
Lingua:Inglês
Pubblicazione: Nature Publishing Group 2010
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Original Articles
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC2889730/
https://ncbi.nlm.nih.gov/pubmed/20389290
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2010.64
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