University of Rochester;University of Florida Research Foundation, Inc.
发明人:
申请号：
EP15186785.0
公开号：
EP3034083A3
申请日:
2007.09.21
申请国别(地区):
EP
年份:
2016
代理人:
摘要：
Disclosed are antisense oligonucleotides for use in treating myotonic dystrophy (DM1 or DM2). The oligonucleotides either inhibit the interaction of poly(CUG)exp or poly(CCUG)exp RNA with muscleblind proteins (MBNL1) or may improve spliceopathy in relation to chloride ion channel ClC-1.
