Correction of metabolic abnormalities in a mouse model of glycogen storage disease type Ia by CRISPR/Cas9-based gene editing

Glycogen storage disease type Ia (GSD-Ia), deficient in glucose-6-phosphatase-α (G6PC), is characterized by impaired glucose homeostasis and a hallmark of fasting hypoglycemia. We have developed a recombinant adeno-associated virus (rAAV) vector-mediated gene therapy for GSD-Ia that is currently in...

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Vydáno v:Mol Ther
Hlavní autoři: Arnaoutova, Irina, Zhang, Lisa, Chen, Hung-Dar, Mansfield, Brian C., Chou, Janice Y.
Médium: Artigo
Jazyk:Inglês
Vydáno: American Society of Gene & Cell Therapy 2021
Témata:
Original Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC8058440/
https://ncbi.nlm.nih.gov/pubmed/33359667
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2020.12.027
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