发明名称 |
SELECTIVE REDUCTION OF THE DELETERIOUS ACTIVITY OF EXTENDED TRI-NUCLEOTIDE REPEAT CONTAINING GENES |
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 |
EP2705151(A2) |
申请公布日期 |
2014.03.12 |
申请号 |
EP20110847833 |
申请日期 |
2011.12.08 |
申请人 |
THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITY;NATIONAL YANG-MING UNIVERSITY |
发明人 |
CHENG, TZU-HAO;LIU, CHIA-RUNG;WANG, TZU-HAN;COHEN, STANLEY N. |
分类号 |
C12N15/113;A61K31/713;A61P25/14 |
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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