Inventors:
Tzu-Hao Cheng - Taipei City, TW
Chia-Rung Liu - Tainan City, TW
Tzu-Han Wang - Taipei City, TW
Stanley N. Cohen - Stanford CA, US
Assignee:
The Board of Trustees of the Leland Stanford Junior University - Palo Alto CA
NATIONAL YANG-MING UNIVERSITY - Taipei
International Classification:
A61K 31/713
US Classification:
514 44 A, 435375, 435 71, 435 611, 435 612
Abstract:
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).