G6PC mRNA Therapy Positively Regulates Fasting Blood Glucose and Decreases Liver Abnormalities in a Mouse Model of Glycogen Storage Disease 1a

Glycogen storage disease type Ia (GSD1a) is an inherited metabolic disorder caused by the deficiency of glucose-6-phosphatase (G6Pase). GSD1a is associated with life-threatening hypoglycemia and long-term liver and renal complications. We examined the efficacy of mRNA-encoding human G6Pase in a live...

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Detalles Bibliográficos
Autores principales: Roseman, Daniel S., Khan, Tayeba, Rajas, Fabienne, Jun, Lucy S., Asrani, Kirtika H., Isaacs, Cleo, Farelli, Jeremiah D., Subramanian, Romesh R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2018
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5910675/
https://www.ncbi.nlm.nih.gov/pubmed/29428299
http://dx.doi.org/10.1016/j.ymthe.2018.01.006

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5910675/
https://www.ncbi.nlm.nih.gov/pubmed/29428299
http://dx.doi.org/10.1016/j.ymthe.2018.01.006

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