University of Rochester;University of Florida Research Foundation, Inc.
发明人:
Charles A. Thornton,Thurman Wheeler,Krzysztof Sobczak,Robert Osborne,Jill Miller,Maurice Scott Swanson
申请号：
US14297137
公开号：
US09371527B2
申请日:
2014.06.05
申请国别(地区):
US
年份:
2016
代理人:
摘要：
Disclosed are compositions and methods related to the interaction of polyCUG and polyCCUG repeat RNA and proteins that bind to these repetitive RNA sequences. Also disclosed are methods of treating DM1 or DM2 comprising inhibiting the interaction of poly(CUG)exp or poly(CCUG)exp RNA with muscleblind proteins, or by causing improvement of spliceopathy in myotonic dystrophy.
