Bezafibrate Enhances AAV Vector-Mediated Genome Editing in Glycogen Storage Disease Type Ia

Glycogen storage disease type Ia (GSD Ia) is a rare inherited disease caused by mutations in the glucose-6-phosphatase (G6Pase) catalytic subunit gene (G6PC). Absence of G6Pase causes life-threatening hypoglycemia and long-term complications because of the accumulations of metabolic intermediates. B...

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Detalles Bibliográficos
Autores principales: Kang, Hye-Ri, Waskowicz, Lauren, Seifts, Andrea M., Landau, Dustin J., Young, Sarah P., Koeberl, Dwight D.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2019
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6395830/
https://www.ncbi.nlm.nih.gov/pubmed/30859111
http://dx.doi.org/10.1016/j.omtm.2019.02.002

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6395830/
https://www.ncbi.nlm.nih.gov/pubmed/30859111
http://dx.doi.org/10.1016/j.omtm.2019.02.002

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