Methods for modulating the expression and aggregation of a CAG-expanded gene product in cells and methods for identifying agents useful for doing the same
Chia-Rung Lu;Stanley N. Cohen;Tzu-Hao Cheng;Tzu-Han Wang
发明人:
Tzu-Hao Cheng,Chia-Rung Lu,Tzu-Han Wang,Stanley N. Cohen
申请号：
US13936696
公开号：
US09226935B2
申请日:
2013.07.08
申请国别(地区):
US
年份:
2016
代理人:
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.
