Pandey, Sanjay K.,Macleod, Robert A.,Swayze, Eric E.,Bennett, C. Frank
申请号：
AU2014306284
公开号：
AU2014306284A1
申请日:
2014.08.11
申请国别(地区):
AU
年份:
2016
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.
