发明名称 |
Methods of Identifying and Using General or Alternative Splicing Inhibitors |
摘要 |
The present invention is based on the discovery of a cell-based system to identify novel modulators of splicing or splicing dependent processes. The cell-based system of the present invention utilizes a fast and highly sensitive reporter, that responds to defects in the splicing machinery itself and is sensitive to changes in the signals that regulate splicing dependent processes such as those that modulate the EJC, splicing-dependent export, localization or translation efficiency. The present invention further uses the cell-based screen to identify several small molecules that modulate both constitutive and alternative splicing. Accordingly, the present invention includes general or alternative splicing inhibitors identified using the assay described herein. The present invention also provides methods of treating a subject having a condition associated with aberrant target RNA expression. The present invention further details a kit that may be directed to specifically detecting a general or alternative splicing inhibitor of the invention.
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申请公布号 |
US2012157474(A1) |
申请公布日期 |
2012.06.21 |
申请号 |
US201013321849 |
申请日期 |
2010.05.21 |
申请人 |
DREYFUSS GIDEON;YOUNIS IHAB;WAN LILI;THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA |
发明人 |
DREYFUSS GIDEON;YOUNIS IHAB;WAN LILI |
分类号 |
A61K31/495;A61K31/155;A61K31/4174;C07C279/26;C07D233/62;C07D241/04;C12N5/071;C12Q1/68;C40B30/04;G01N21/75 |
主分类号 |
A61K31/495 |
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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