Genome editing using Staphylococcus aureus Cas9 in a canine model of glycogen storage disease Ia

Genome editing using Staphylococcus aureus Cas9 in a canine model of glycogen storage disease Ia

Glycogen storage disease type Ia (GSD Ia) is the inherited deficiency of glucose-6-phosphatase (G6Pase), associated with life-threatening hypoglycemia and long-term complications, including hepatocellular carcinoma formation. Gene replacement therapy fails to stably reverse G6Pase deficiency. We att...

Full description

Bibliographic Details
Main Authors: Benjamin Arnson, Hye Ri Kang, Elizabeth D. Brooks, Dorothy Gheorghiu, Ekaterina Ilich, David Courtney, Jeffrey I. Everitt, Bryan R. Cullen, Dwight D. Koeberl
Format: Article
Language:English
Published: Elsevier 2023-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
genome editing
CRISPR-Cas9
adeno-associated virus vectors
inherited disorder of metabolism
glycogen storage disease
glucose-6-phosphatase
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050123000372

Internet

http://www.sciencedirect.com/science/article/pii/S2329050123000372

Similar Items