THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITY;NATIONAL YANG-MING UNIVERSITY
发明人:
COHEN, STANLEY N.,DENG, NING,FENG, YANAN,CHENG, TZU-HAO,WU, YUN-YUN,HSIEH, WEN-CHIEH
申请号：
CA2978521
公开号：
CA2978521A1
申请日:
2016.05.18
申请国别(地区):
CA
年份:
2016
代理人:
摘要：
Aspects of the invention include methods of reducing the deleterious activity of a mutant extended nucleotide repeat (NR) containing target gene in a cell by contacting the cell with an effective amount of a nucleoside agent, 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 NR containing target gene may be reduced in a variety of different ways, e.g., by reducing (and in some instances differentially, including selectively, reducing) the production or activity of toxic expression products (e.g., RNA or protein) encoded by the target gene. Kits and compositions for practicing the subject methods are also provided.
