The Board of Regents of the University of Texas System
发明人:
OLSON, Eric N.,LONG, Chengzu
申请号：
AU2019216321
公开号：
AU2019216321A1
申请日:
2019.01.31
申请国别(地区):
AU
年份:
2020
代理人:
摘要：
The disclosure provides a method for treating or preventing Duchene Muscular Dystrophy (DMD) in a subject in need thereof, the method comprising administering to the subject a Cas9 nuclease or a sequence encoding a Cas9 nuclease, and a gRNA or a sequence encoding a gRNA, wherein the gRNA targets a splice donor or splice acceptor site of the dystrophin gene. The administering restores dystrophin expression in at least a subset of the subject's cardiomyocytes, and may at least partially or fully restore cardiac contractility.
