发明名称 |
COMPOSITIONS AND METHODS FOR MODULATION OF SMN2 SPLICING |
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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申请公布号 |
US2010216238(A1) |
申请公布日期 |
2010.08.26 |
申请号 |
US20060993609 |
申请日期 |
2006.06.23 |
申请人 |
ISIS PHARMACEUTICALS, INC. |
发明人 |
BAKER BRENDA F;KRAINER ADRIAN R.;HUA YIMIN |
分类号 |
C12N5/02;C07H21/04 |
主分类号 |
C12N5/02 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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