	US 12,419,970 B2
	Gene therapy for diseases caused by unbalanced nucleotide pools including mitochondrial DNA depletion syndromes
Michio Hirano, New York, NY (US); Hasan O. Akman, Haworth, NJ (US); and Carlos Lopez-Gomez, Fuengirola (ES)
Assigned to The Trustees of Columbia University in the City of New York, New York, NY (US)
Appl. No. 17/048,236
Filed by The Trustees of Columbia University in the City of New York, New York, NY (US)
PCT Filed Apr. 18, 2019, PCT No. PCT/US2019/028108 § 371(c)(1), (2) Date Oct. 16, 2020, PCT Pub. No. WO2019/204593, PCT Pub. Date Oct. 24, 2019.
Claims priority of provisional application 62/659,281, filed on Apr. 18, 2018.
Prior Publication US 2021/0100917 A1, Apr. 8, 2021
Int. Cl. A61K 38/45 (2006.01); A61K 48/00 (2006.01); C07K 14/705 (2006.01); C12N 15/86 (2006.01)

CPC A61K 48/005 (2013.01) [A61K 38/45 (2013.01); C07K 14/705 (2013.01); C12N 15/86 (2013.01); C12N 2750/14143 (2013.01); C12Y 207/01021 (2013.01); C12Y 207/01076 (2013.01); C12Y 207/01113 (2013.01)]

6 Claims

1. A method of increasing weight gain in a subject suffering from myopathic MDS characterized by one or more mutations in an endogenous gene encoding TK2, the method comprising administering to the subject a therapeutically effective amount of a composition comprising a vector of adeno-associated virus (AAV) and comprising a transgene encoding thymidine kinase 2 (TK2).