The Board of Trustees of the Leland Stanford Junior University;National Yang-Ming University
发明人:
Tzu-Hao Cheng,Chia-Rung Liu,Tzu-Han Wang,Stanley N. Cohen
申请号：
US15833891
公开号：
US20180171336A1
申请日:
2017.12.06
申请国别(地区):
US
年份:
2018
代理人:
摘要：
Aspects of the invention include methods of selectively reducing the deleterious activity of mutant extended trinucleotide repeat containing genes in a cell, as well as compositions used in such methods. The deleterious activity (e.g., toxicity and/or dis-functionality of products encoded thereby) of a mutant extended trinucleotide repeat containing gene may be selectively reduced in a variety of different ways, e.g., by selectively decreasing SPT4 mediated transcriptional activity, by enhancing functionality of proteins encoded thereby, etc. Aspects of the invention further include assays for identifying agents that find use in methods of the invention, e.g. as summarized above. Methods and compositions of the invention find use in a variety of different applications, including the prevention or treatment of disease conditions associated with the presence of genes containing mutant extended trinucleotide repeats, such as Huntington's Disease (HD).
