- 专利标题： COMPOSITIONS AND METHODS FOR MODULATION OF SMN2 SPLICING
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申请号： US15267408申请日： 2016-09-16
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公开(公告)号： US20170088835A1公开(公告)日： 2017-03-30
- 发明人: Brenda F. Baker , Adrian R. Krainer , Yimin Hua
- 申请人： Biogen MA Inc. , Cold Spring Harbor Laboratory
- 申请人地址： US MA Cambridge US NY Cold Spring Harbor
- 专利权人： Biogen MA Inc.,Cold Spring Harbor Laboratory
- 当前专利权人： Biogen MA Inc.,Cold Spring Harbor Laboratory
- 当前专利权人地址： US MA Cambridge US NY Cold Spring Harbor
- 主分类号： C12N15/113
- IPC分类号： C12N15/113
摘要：
Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a cell, tissue or animal. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy.
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