Janice J CHOU;THE UNITED STATES OF AMERICA as represented by THE SECRETARY DEPARTMENT OF HEALTH AND HUMAN SERVICES
发明人:
Janice J CHOU
申请号：
IL25310317
公开号：
IL253103A
申请日:
2017.06.22
申请国别(地区):
IL
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.
