THE BOARD OF REGENTS OF THE UNIVERSITY OF TEXAS SYSTEM
发明人:
OLSON, ERIC N.,LONG, CHENGZU
申请号：
CA3088547
公开号：
CA3088547A1
申请日:
2019.01.31
申请国别(地区):
CA
年份:
2019
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.
