Compositions and methods related to protein displacement therapy for myotonic distrophy
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.
申请公布号
EP2560001(B1)
申请公布日期
2016.04.13
申请号
EP20120005320
申请日期
2007.09.21
申请人
UNIVERSITY OF ROCHESTER;UNIVERSITY OF FLORIDA RESEARCH FOUNDATION, INC.
发明人
THORNTON, CHARLES, A.;WHEELER, THURMAN;SOBEZAK, KRZYSZTOF;OSBORNE, ROBERT;MILLER, JILL;SWANSON, MAURICE, S.