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Gene therapy prevents hepatic tumor initiation in murine glycogen storage disease type Ia at the tumor-developing stage

Hepatocellular adenoma/carcinoma (HCA/HCC) is a long-term complication of glycogen storage disease type-Ia (GSD-Ia) which is caused by a deficiency in glucose-6-phosphatase-α (G6Pase-α or G6PC), a key enzyme in gluconeogenesis. Currently, there is no therapy to address HCA/HCC in GSD-Ia. We have pre...

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Detalhes bibliográficos
Publicado no:J Inherit Metab Dis
Main Authors: Cho, Jun-Ho, Lee, Young Mok, Starost, Matthew F., Mansfield, Brian C., Chou, Janice Y.
Formato: Artigo
Idioma:Inglês
Publicado em: 2019
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6483852/
https://ncbi.nlm.nih.gov/pubmed/30637773
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/jimd.12056
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