Design of a bioactive small molecule that targets r(AUUCU) repeats in spinocerebellar ataxia 10
Wang-Yong Yang, Rui Gao, Mark Southern and 2 more
Nature Communications · 2016
doi:10.1038/ncomms11647 (opens in a new tab)
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Abstract
Expanded RNA repeats in non-coding region of a gene represent a hallmark of several diseases. Here, the authors identify two small molecules that selectively bind AU repeats and use them to design a compound that targets the pathogenic RNA associated with spinocerebellar ataxia type 10.
Abstract licence: CC BY-NC-ND
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