THE GOVERNMENT OF 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：
CA3050917
公开号：
CA3050917A1
申请日:
2018.01.30
申请国别(地区):
CA
年份:
2018
代理人:
摘要：
Recombinant viruses, such as adeno-associated virus (rAAV) or lentivirus, for the treatment of glycogen storage disease type lb (GSD-Ib) are described. The recombinant viruses use either the human glucose-6-phosphatase (G6PC) promoter/enhancer (GPE) or the minimal human G6PT promoter/enhancer (miGT) to drive expression of human glucose-6-phosphate transporter (G6PT). The disclosed vectors are capable of delivering the G6PT transgene to the liver and correcting metabolic abnormalities in a murine model of GSD-Ib. The recombinant virus-treated mice maintained glucose homeostasis, tolerated a long fast, and did not elicit anti-G6PT antibodies. Methods of treating a subject diagnosed with GSD-Ib using the recombinant viruses is further described.
