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Pathogenesis of Hepatic Tumors following Gene Therapy in Murine and Canine Models of Glycogen Storage Disease

Glycogen storage disease type Ia (GSD Ia) is caused by mutations in the glucose-6-phosphatase (G6Pase) catalytic subunit gene (G6PC). GSD Ia complications include hepatocellular adenomas (HCA) with a risk for hepatocellular carcinoma (HCC) formation. Genome editing with adeno-associated virus (AAV)...

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Detalles Bibliográficos
Autores principales: Kang, Hye-Ri, Gjorgjieva, Monika, Smith, Stephanie N., Brooks, Elizabeth D., Chen, Zelin, Burgess, Shawn M., Chandler, Randy J., Waskowicz, Lauren R., Grady, Kylie M., Li, Songtao, Mithieux, Gilles, Venditti, Charles P., Rajas, Fabienne, Koeberl, Dwight D.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6909089/
https://www.ncbi.nlm.nih.gov/pubmed/31890731
http://dx.doi.org/10.1016/j.omtm.2019.10.016