发明名称 |
Methods for modulating the expression and aggregation of CAG-expanded gene product in cells and methods for identifying agents useful for doing the same |
摘要 |
This invention provides a method for modulating the expression of a first gene in a cell wherein the first gene is one containing more than 36 CAG trinucleotide repeats and encoding a protein that form polyglutamine-mediated protein aggregation. Suppression of the first gene is achieved by reducing the expression of SPT4 gene or SUPT4H gene. It can also be achieved by inhibiting the formation of a Spt4/Spt5 complex or a Supt4h/Supt5h complex. Also provided is a method for identifying an agent useful for modulating the expression and aggregation of CAG-expanded gene product, or treating a polyglutamine disease such as Huntington's disease. |
申请公布号 |
EP2463372(B1) |
申请公布日期 |
2015.09.02 |
申请号 |
EP20110167774 |
申请日期 |
2011.05.26 |
申请人 |
NATIONAL YANG-MING UNIVERSITY |
发明人 |
CHENG, TZU-HAO;LIU, CHIA-RUNG;WANG, TZU-HAN;COHEN, STANLEY N. |
分类号 |
C12N15/113;G01N33/50 |
主分类号 |
C12N15/113 |
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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