发明名称 |
COMPOSITIONS AND METHODS FOR MODULATION OF SMN2 SPLICING |
摘要 |
<p>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.</p> |
申请公布号 |
PT2548560(E) |
申请公布日期 |
2015.09.22 |
申请号 |
PT20060188625T |
申请日期 |
2006.06.23 |
申请人 |
ISIS PHARMACEUTICALS, INC. |
发明人 |
BRENDA F. BAKER;ADRIAN R. KRAINER;YIMIN HUA |
分类号 |
A61K31/70;C12N15/11;C12N15/113 |
主分类号 |
A61K31/70 |
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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