| 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 |
US2017088835(A1) |
申请公布日期 |
2017.03.30 |
| 申请号 |
US201615267408 |
申请日期 |
2016.09.16 |
| 申请人 |
Biogen MA 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 |
1. An antisense oligonucleotide targeted to intron 6, exon 7 or intron 7 of a nucleic acid molecule encoding SMN2, wherein said oligonucleotide is 12 to 20 nucleotides in length and comprises a 2′-O-methoxyethyl sugar modification at each position. |
| 地址 |
Cambridge MA US |
