| 发明名称 |
COMPOSITIONS AND METHODS FOR TREATING AMYOTROPHIC LATERAL SCLEROSIS |
| 摘要 |
Methods for modulating expression of SMN1 and/or SMN2 in cells obtained from subjects having ALS or in subjects having ALS using single stranded oligonucleotides are provided. Methods for treating ALS using single stranded oligonucleotides are also provided. |
| 申请公布号 |
EP3052632(A4) |
申请公布日期 |
2017.03.29 |
| 申请号 |
EP20140851110 |
申请日期 |
2014.10.03 |
| 申请人 |
Rana Therapeutics, Inc. |
发明人 |
KRIEG, Arthur, M.;BARSOUM, James |
| 分类号 |
C12N15/63;A61K31/7088;A61P21/00;C12N5/0793 |
主分类号 |
C12N15/63 |
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