US 12,186,369 B2
Gene therapy strategy to restore electrical and cardiac function, and cardiac structure, in arrhythmogenic right ventricular cardiomyopathy
Farah Sheikh, La Jolla, CA (US); and Jing Zhang, La Jolla, CA (US)
Assigned to The Regents of the University of California, Oakland, CA (US)
Appl. No. 16/648,922
Filed by THE REGENTS OF THE UNIVERSITY OF CALIFORNIA, Oakland, CA (US)
PCT Filed Sep. 20, 2018, PCT No. PCT/US2018/052057
§ 371(c)(1), (2) Date Mar. 19, 2020,
PCT Pub. No. WO2019/060619, PCT Pub. Date Mar. 28, 2019.
Claims priority of provisional application 62/560,989, filed on Sep. 20, 2017.
Prior Publication US 2020/0215155 A1, Jul. 9, 2020
Int. Cl. C12N 15/00 (2006.01); A61K 35/761 (2015.01); A61K 38/17 (2006.01); A61P 9/06 (2006.01); C12N 7/00 (2006.01); C12N 15/86 (2006.01); A61K 48/00 (2006.01)
CPC A61K 38/177 (2013.01) [A61K 35/761 (2013.01); A61P 9/06 (2018.01); C12N 7/00 (2013.01); C12N 15/86 (2013.01); A61K 48/00 (2013.01); C12N 2710/10343 (2013.01); C12N 2750/14143 (2013.01)] 12 Claims
OG exemplary drawing
 
1. A method for treating arrhythmogenic right ventricular cardiomyopathy (ARVC) in a subject in need thereof, comprising systemically administering to the subject an adeno-associated virus (AAV) vector encoding a connexin 43 polypeptide sequence operably linked to a promoter that is active in cardiac muscle tissue such that connexin 43 polypeptide levels in at least a portion of the heart of the subject are increased.