	US 12,233,050 B2
	Drug and gene therapy to treat high myopia and other ocular disorders with enlarged eye globes
Wenjun Xiong, Kowloon (HK); and Shuyi Mai, Kowloon (HK)
Assigned to CITY UNIVERSITY OF HONG KONG, Kowloon (HK)
Filed by City University of Hong Kong, Kowloon (HK)
Filed on Jun. 11, 2021, as Appl. No. 17/346,036.
Claims priority of provisional application 63/038,163, filed on Jun. 12, 2020.
Prior Publication US 2021/0386714 A1, Dec. 16, 2021
Int. Cl. C12N 15/86 (2006.01); A61K 31/21 (2006.01); A61K 31/426 (2006.01); A61K 48/00 (2006.01); C07H 21/04 (2006.01); C12N 15/864 (2006.01)

CPC A61K 31/426 (2013.01) [A61K 31/21 (2013.01); A61K 48/0058 (2013.01); C12N 15/86 (2013.01); C07H 21/04 (2013.01); C12N 15/8645 (2013.01); C12N 2750/14143 (2013.01)]

3 Claims

1. A method of treating Donnai-Barrow syndrome in a subject in need thereof, comprising a step of administering to the subject a recombinant adeno-associated virus (AAV) vector comprising a nucleic acid encoding a normal bone morphogenetic protein 2 (BMP2) gene operably linked to a human Bestrophin1 (hBEST1) promoter and a Simian virus 40 (SV40) intron, wherein the AAV vector is administered by subretinal injection and can increase the expression of BMP2 in the subject, and rescue eye enlargement phenotype.