Design of a bioactive small molecule that targets r(AUUCU) repeats in spinocerebellar ataxia 10


RNA is an important target for chemical probes of function and lead therapeutics; however, it is difficult to target with small molecules. One approach to tackle this problem is to identify compounds that target RNA structures and utilize them to multivalently target RNA. Here we show that small molecules can be identified to selectively bind RNA base pairs… (More)
DOI: 10.1038/ncomms11647