发明公开
EP2560001A3 Compositions and methods related to protein displacement therapy for myotonic distrophy
有权
Zusammensetzungen und Verfahrenfüreine Proteinverlagerungstherapiefürmyotone Dystrophie
- 专利标题： Compositions and methods related to protein displacement therapy for myotonic distrophy
- 专利标题（中）： Zusammensetzungen und Verfahrenfüreine Proteinverlagerungstherapiefürmyotone Dystrophie
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申请号： EP12005320.2申请日： 2007-09-21
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公开(公告)号： EP2560001A3公开(公告)日： 2013-07-17
- 发明人: Thornton, Charles, A. , Wheeler, Thurman , Sobezak, Krzysztof , Osborne, Robert , Miller, Jill , Swanson, Maurice, S.
- 申请人： University of Rochester , UNIVERSITY OF FLORIDA RESEARCH FOUNDATION, INC.
- 申请人地址： 601 Elmwood Avenue, Box OTT/OCA Rochester, NY 14642 US
- 专利权人： University of Rochester,UNIVERSITY OF FLORIDA RESEARCH FOUNDATION, INC.
- 当前专利权人： University of Rochester,UNIVERSITY OF FLORIDA RESEARCH FOUNDATION, INC.
- 当前专利权人地址： 601 Elmwood Avenue, Box OTT/OCA Rochester, NY 14642 US
- 代理机构： UEXKÜLL & STOLBERG
- 优先权： US826396 20060921
- 主分类号： G01N33/50
- IPC分类号： G01N33/50 ; G01N33/68 ; C12Q1/68 ; C12N15/113 ; A61K31/712 ; A61K31/7125 ; A61K31/7115 ; A61K31/5375 ; A61K31/7036
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.
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