Ionis Pharmaceuticals, Inc.;Baylor College Of Medicine
发明人:
RIGO, Frank,WARD, Amanda,BEAUDET, Arthur L.,MENG, Linyan
申请号:
DK18188705
公开号:
DK3461895T3
申请日:
2013.06.25
申请国别(地区):
DK
年份:
2020
代理人:
摘要:
Certain embodiments are directed to methods and compounds for inhibiting UBE3A-ATS, the endogenous antisense transcript of ubiquitin protein ligase E3A (UBE3A). Such methods and compounds are useful for inducing expression of paternal UBE3A in cells and animals.