发明公开
EP3031920A1 MODULATION OF DYSTROPHIA MYOTONICA-PROTEIN KINASE (DMPK) EXPRESSION
审中-公开
调节剂DYSTROPHIA-MYOTONICA-PROTEINKINASE（DMPK）-EXPRESSION
- 专利标题： MODULATION OF DYSTROPHIA MYOTONICA-PROTEIN KINASE (DMPK) EXPRESSION
- 专利标题（中）： 调节剂DYSTROPHIA-MYOTONICA-PROTEINKINASE（DMPK）-EXPRESSION
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申请号： EP16153949.9申请日： 2011-07-19
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公开(公告)号： EP3031920A1公开(公告)日： 2016-06-15
- 发明人: BENNETT, C. Frank , FREIER, Susan M. , MACLEOD, Robert A. , PANDEY, Sanjey K. , THORNTON, Charles A. , WHEELER, Thurman , CHENG, Seng H. , LEGER, Andrew , WENTWORTH, Bruce M.
- 申请人： Ionis Pharmaceuticals, Inc. , University of Rochester
- 申请人地址： 2855 Gazelle Court Carlsbad, CA 92010 US
- 专利权人： Ionis Pharmaceuticals, Inc.,University of Rochester
- 当前专利权人： Ionis Pharmaceuticals, Inc.,University of Rochester
- 当前专利权人地址： 2855 Gazelle Court Carlsbad, CA 92010 US
- 代理机构： Carpmaels & Ransford LLP
- 优先权： US365762P 20100719; US365775P 20100719; US201161478021P 20110421
- 主分类号： C12N15/113
- IPC分类号： C12N15/113 ; A61K31/7088 ; A61P21/00
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 spliceopathy in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate type 1 myotonic dystrophy, or a symptom thereof.
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