| 发明名称 |
Methods of screening for compounds for treating spinal muscular atrophy using SMN mRNA translation regulation |
| 摘要 |
The present invention provides compounds and assays for the identification and validation of compounds for use in the treatment of spinal muscular atrophy (SMA), in which said compounds up-regulate the post-transcriptional expression of SMN1 or SMN2. |
| 申请公布号 |
US8283116(B1) |
申请公布日期 |
2012.10.09 |
| 申请号 |
US20080144577 |
申请日期 |
2008.06.23 |
| 申请人 |
PTC THERAPEUTICS, INC. |
发明人 |
BHATTACHARYYA ANURADHA;FURIA BANSRI S.;PATEL MEENAL;PAUSHKIN SERGEY V.;VOLKOVA LOVE |
| 分类号 |
C12Q1/68 |
主分类号 |
C12Q1/68 |
| 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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