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Liver-directed gene therapy for murine glycogen storage disease type Ib

Glycogen storage disease type-Ib (GSD-Ib), deficient in the glucose-6-phosphate transporter (G6PT), is characterized by impaired glucose homeostasis, myeloid dysfunction, and long-term risk of hepatocellular adenoma (HCA). We examined the efficacy of G6PT gene therapy in G6pt−/− mice using recombina...

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Detalhes bibliográficos
Publicado no:Hum Mol Genet
Main Authors: Kwon, Joon Hyun, Lee, Young Mok, Cho, Jun-Ho, Kim, Goo-Young, Anduaga, Javier, Starost, Matthew F, Mansfield, Brian C, Chou, Janice Y
Formato: Artigo
Idioma:Inglês
Publicado em: Oxford University Press 2017
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5886224/
https://ncbi.nlm.nih.gov/pubmed/28973635
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddx325
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