发明申请
US20120149754A1 METHODS FOR MODULATING THE EXPRESSION AND AGGREGATION OF CAG-EXPANDED GENE PRODUCT IN CELLS AND METHODS FOR IDENTIFYING AGENTS USEFUL FOR DOING THE SAME
有权
用于调节细胞中CAG扩增的基因产物的表达和聚集的方法以及用于识别有用的代谢物的方法
- 专利标题： METHODS FOR MODULATING THE EXPRESSION AND AGGREGATION OF CAG-EXPANDED GENE PRODUCT IN CELLS AND METHODS FOR IDENTIFYING AGENTS USEFUL FOR DOING THE SAME
- 专利标题（中）： 用于调节细胞中CAG扩增的基因产物的表达和聚集的方法以及用于识别有用的代谢物的方法
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申请号： US13110494申请日： 2011-05-18
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公开(公告)号： US20120149754A1公开(公告)日： 2012-06-14
- 发明人: Tzu-Hao CHENG , Chia-Rung LIU , Tzu-Han WANG
- 申请人： Tzu-Hao CHENG , Chia-Rung LIU , Tzu-Han WANG
- 申请人地址： TW Taipei
- 专利权人： NATIONAL YANG MING UNIVERSITY
- 当前专利权人： NATIONAL YANG MING UNIVERSITY
- 当前专利权人地址： TW Taipei
- 优先权： TW099143336 20101210
- 主分类号： A61K31/7088
- IPC分类号： A61K31/7088 ; C12N15/85 ; A61P25/00 ; C12N1/14 ; C12N5/071 ; C12Q1/68 ; C12N15/81 ; C12N5/07
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.