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

Autor: Wang-Yong Yang, Rui Gao, Mark Southern, Partha S. Sarkar, Matthew D. Disney
Jazyk: angličtina
Rok vydání: 2016
Předmět:
Zdroj: Nature Communications, Vol 7, Iss 1, Pp 1-11 (2016)
Druh dokumentu: article
ISSN: 2041-1723
DOI: 10.1038/ncomms11647
Popis: 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.
Databáze: Directory of Open Access Journals