US 12,440,580 B2
Adeno-associated virus vectors encoding modified G6PC and uses thereof
Janice J. Chou, North Bethesda, MD (US)
Assigned to The United States of America, as represented by the Secretary, Department of Health and Human Services, Bethesda, MD (US)
Filed by The U.S.A., as represented by the Secretary, Department of Health and Human Services, Bethesda, MD (US)
Filed on Nov. 23, 2022, as Appl. No. 18/058,457.
Application 18/058,457 is a division of application No. 16/526,327, filed on Jul. 30, 2019, granted, now 11,535,870.
Application 16/526,327 is a division of application No. 15/538,852, granted, now 10,415,044, issued on Sep. 17, 2019, previously published as PCT/US2015/067338, filed on Dec. 22, 2015.
Claims priority of provisional application 62/096,400, filed on Dec. 23, 2014.
Prior Publication US 2023/0144834 A1, May 11, 2023
Int. Cl. C12N 15/86 (2006.01); A61K 38/00 (2006.01); A61K 38/47 (2006.01); A61K 48/00 (2006.01); C12N 9/16 (2006.01); C12N 15/66 (2006.01); C12N 15/85 (2006.01); C12N 15/864 (2006.01); A61K 45/06 (2006.01)
CPC A61K 48/0075 (2013.01) [A61K 38/00 (2013.01); A61K 38/47 (2013.01); A61K 48/00 (2013.01); C12N 9/16 (2013.01); C12N 15/66 (2013.01); C12N 15/85 (2013.01); C12N 15/8645 (2013.01); C12Y 301/03009 (2013.01); A61K 45/06 (2013.01); C12Y 302/00 (2013.01)] 11 Claims
 
1. A method of promoting glucose homeostasis in a subject diagnosed with glycogen storage disease type Ia (GSD-Ia), comprising:
selecting a subject with GSD-Ia; and
intravenously administering to the subject a therapeutically effective amount of a recombinant AAV (rAAV) comprising a nucleic acid molecule encoding a modified glucose-6-phosphatase-α (G6Pase-α) operably linked to a promoter, wherein the modified G6Pase-α comprises a serine to cysteine substitution at amino acid 298 of human G6Pase-α set forth as SEQ ID NO: 2.