THE UNITED STATES OF AMERICA; AS REPRESENTED BY THE SECRETARY; DEPARTMENT OF HEALTH AND HUMAN SERVICES
发明人:
CHOU, JANICE J.
申请号：
CA2972038
公开号：
CA2972038A1
申请日:
2015.12.22
申请国别(地区):
CA
年份:
2016
代理人:
摘要：
Modified G6PC (glucose-6-phosphatase, catalytic subunit) nucleic acids and glucose-6- phosphatase-a (G6Pase-a) enzymes with increased phosphohydrolase activity are described. Also described are vectors, such as adeno-associated virus (AAV) vectors, and recombinant AAV expressing modified G6Pase-a. 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 la (GSD-Ia), and complications thereof.
