Article
Transcriptional Inhibitors Identified in a 160,000-Compound Small-Molecule DUX4 Viability Screen.
Journal of biomolecular screening - 1 Aug 2016
Choi Si Ho, Bosnakovski Darko, Strasser Jessica M, Toso Erik A, Walters Michael A, Kyba Michael
Abstract excerpt
Facioscapulohumeral muscular dystrophy is a genetically dominant, currently untreatable muscular dystrophy. It is caused by mutations that enable expression of the normally silent DUX4 gene, which encodes a pathogenic transcription factor. A screen based on Tet-on DUX4-induced mouse myoblast death previously uncovered compounds from a 44,000-compound library that protect against DUX4 toxicity. Many of those...
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