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

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

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.

Bibliographic Details
Main Authors: Wang-Yong Yang, Rui Gao, Mark Southern, Partha S. Sarkar, Matthew D. Disney
Format: Article
Language:English
Published: Nature Publishing Group 2016-06-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/ncomms11647
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spelling doaj-f56a67cc0b8b4f9395ec9cf1058103142021-05-11T11:02:37ZengNature Publishing GroupNature Communications2041-17232016-06-017111110.1038/ncomms11647Design of a bioactive small molecule that targets r(AUUCU) repeats in spinocerebellar ataxia 10Wang-Yong Yang0Rui Gao1Mark Southern2Partha S. Sarkar3Matthew D. Disney4Departments of Chemistry and Neuroscience, The Scripps Research Institute, Scripps FloridaDepartment of Neurology, Mitchell Center for Neurodegenerative Disorders, Neuroscience and Cell Biology, University of Texas Medical BranchInformatics Core, The Scripps Research Institute, Scripps FloridaDepartment of Neurology, Mitchell Center for Neurodegenerative Disorders, Neuroscience and Cell Biology, University of Texas Medical BranchDepartments of Chemistry and Neuroscience, The Scripps Research Institute, Scripps FloridaExpanded 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.https://doi.org/10.1038/ncomms11647
collection DOAJ
language English
format Article
sources DOAJ
author Wang-Yong Yang
Rui Gao
Mark Southern
Partha S. Sarkar
Matthew D. Disney
spellingShingle Wang-Yong Yang
Rui Gao
Mark Southern
Partha S. Sarkar
Matthew D. Disney
Design of a bioactive small molecule that targets r(AUUCU) repeats in spinocerebellar ataxia 10
Nature Communications
author_facet Wang-Yong Yang
Rui Gao
Mark Southern
Partha S. Sarkar
Matthew D. Disney
author_sort Wang-Yong Yang
title Design of a bioactive small molecule that targets r(AUUCU) repeats in spinocerebellar ataxia 10
title_short Design of a bioactive small molecule that targets r(AUUCU) repeats in spinocerebellar ataxia 10
title_full Design of a bioactive small molecule that targets r(AUUCU) repeats in spinocerebellar ataxia 10
title_fullStr Design of a bioactive small molecule that targets r(AUUCU) repeats in spinocerebellar ataxia 10
title_full_unstemmed Design of a bioactive small molecule that targets r(AUUCU) repeats in spinocerebellar ataxia 10
title_sort design of a bioactive small molecule that targets r(auucu) repeats in spinocerebellar ataxia 10
publisher Nature Publishing Group
series Nature Communications
issn 2041-1723
publishDate 2016-06-01
description 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.
url https://doi.org/10.1038/ncomms11647
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AT ruigao designofabioactivesmallmoleculethattargetsrauucurepeatsinspinocerebellarataxia10
AT marksouthern designofabioactivesmallmoleculethattargetsrauucurepeatsinspinocerebellarataxia10
AT parthassarkar designofabioactivesmallmoleculethattargetsrauucurepeatsinspinocerebellarataxia10
AT matthewddisney designofabioactivesmallmoleculethattargetsrauucurepeatsinspinocerebellarataxia10
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