PANDEY, SANJAY K.,MACLEOD, ROBERT A.,SWAYZE, ERIC E.,BENNETT, C. FRANK
申请号：
TW103127342
公开号：
TW201536329A
申请日:
2014.08.08
申请国别(地区):
TW
年份:
2015
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 spliceopathyin an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate type 1 myotonic dystrophy, or a symptom thereof.本文提供用於降低動物中之DMPK mRNA及蛋白質之表現的方法、化合物及組合物。本文亦提供用於在動物中優先降低CUGexp DMPK RNA、減輕肌強直或減輕拼接病變之方法、化合物及組合物。此等方法、化合物及組合物適用於治療、預防、延遲或改善1型肌強直性營養不良或其症狀。
