The U.S.A.; as represented by the Secretary; Department of Health and Human Services
发明人:
Janice J. Chou
申请号：
US16526327
公开号：
US20190345502A1
申请日:
2019.07.30
申请国别(地区):
US
年份:
2019
代理人:
摘要：
Modified G6PC (glucose-6-phosphatase, catalytic subunit) nucleic acids and glucose-6-phosphatase-α (G6Pase-α) enzymes with increased phosphohydrolase activity are described. Also described are vectors, such as adeno-associated virus (AAV) vectors, and recombinant AAV expressing modified G6Pase-α. The disclosed AAV vectors and rAAV can be used for gene therapy applications in the treatment of glycogen storage disease, particularly glycogen storage disease type Ia (GSD-Ia), and complications thereof.
