Ionis Pharmaceuticals, Inc.;The Regents of the University of California;Ludwig Institute for Cancer Research
发明人:
C. Frank Bennett,Frank Rigo,Don W. Cleveland,Clotilde Lagier-Tourenne,John M. Ravits,Michael W. Baughn
申请号：
US15848389
公开号：
US20180142240A1
申请日:
2017.12.20
申请国别(地区):
US
年份:
2018
代理人:
摘要：
Disclosed herein are compositions and methods for reducing expression of C9ORF72 antisense transcript in an animal with C9ORF72 antisense transcript specific inhibitors. Such methods are useful to treat, prevent, or ameliorate neurodegenerative diseases in an individual in need thereof. Such C9ORF72 antisense transcript specific inhibitors include antisense compounds.
