Ionis Pharmaceuticals, Inc.;University of Rochester
发明人:
BENNETT, C. FRANK,FREIER, SUSAN M.,MACLEOD, ROBERT A.,PANDEY, SANJEY K.,THORNTON, CHARLES A.,WHEELER, THURMAN,CHENG, SENG H.,LEGER, ANDREW,WENTWORTH, BRUCE M.
申请号：
PL16153949
公开号：
PL3031920T3
申请日:
2011.07.19
申请国别(地区):
PL
年份:
2020
代理人:
摘要：
Provided herein are methods, compounds, and compositions for reducing expression of a DMPK mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions for preferentially reducing CUGexp DMPK RNA, reducing myotonia or reducing spliceopathy in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate type 1 myotonic dystrophy, or a symptom thereof.
