| 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. |
| 申请公布号 |
US2015353929(A1) |
申请公布日期 |
2015.12.10 |
| 申请号 |
US201414584112 |
申请日期 |
2014.12.29 |
| 申请人 |
Isis Pharmaceuticals, Inc. ;Cold Spring Harbor Laboratory |
发明人 |
Baker Brenda F.;Krainer Adrian R.;Hua Yimin |
| 分类号 |
C12N15/113 |
主分类号 |
C12N15/113 |
| 代理机构 |
|
代理人 |
|
| 主权项 |
|
| 地址 |
Carlsbad CA US |
