Small molecule targeting r(UGGAA) n disrupts RNA foci and alleviates disease phenotype in Drosophila model

Synthetic small molecules modulating RNA structure and function have therapeutic potential for RNA diseases. Here the authors show the mechanism by which a small molecule targets the disease-causing r(UGGAA) n repeat RNAs in spinocerebellar ataxia type 31.

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Autores principales:	Tomonori Shibata, Konami Nagano, Morio Ueyama, Kensuke Ninomiya, Tetsuro Hirose, Yoshitaka Nagai, Kinya Ishikawa, Gota Kawai, Kazuhiko Nakatani
Formato:	article
Lenguaje:	EN
Publicado:	Nature Portfolio 2021
Materias:	Science Q
Acceso en línea:	https://doaj.org/article/fb74152ab4824a57b44c57a7b829b312
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spelling	oai:doaj.org-article:fb74152ab4824a57b44c57a7b829b3122021-12-02T15:22:39ZSmall molecule targeting r(UGGAA) n disrupts RNA foci and alleviates disease phenotype in Drosophila model10.1038/s41467-020-20487-42041-1723https://doaj.org/article/fb74152ab4824a57b44c57a7b829b3122021-01-01T00:00:00Zhttps://doi.org/10.1038/s41467-020-20487-4https://doaj.org/toc/2041-1723Synthetic small molecules modulating RNA structure and function have therapeutic potential for RNA diseases. Here the authors show the mechanism by which a small molecule targets the disease-causing r(UGGAA) n repeat RNAs in spinocerebellar ataxia type 31.Tomonori ShibataKonami NaganoMorio UeyamaKensuke NinomiyaTetsuro HiroseYoshitaka NagaiKinya IshikawaGota KawaiKazuhiko NakataniNature PortfolioarticleScienceQENNature Communications, Vol 12, Iss 1, Pp 1-13 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	Science Q
spellingShingle	Science Q Tomonori Shibata Konami Nagano Morio Ueyama Kensuke Ninomiya Tetsuro Hirose Yoshitaka Nagai Kinya Ishikawa Gota Kawai Kazuhiko Nakatani Small molecule targeting r(UGGAA) n disrupts RNA foci and alleviates disease phenotype in Drosophila model
description	Synthetic small molecules modulating RNA structure and function have therapeutic potential for RNA diseases. Here the authors show the mechanism by which a small molecule targets the disease-causing r(UGGAA) n repeat RNAs in spinocerebellar ataxia type 31.
format	article
author	Tomonori Shibata Konami Nagano Morio Ueyama Kensuke Ninomiya Tetsuro Hirose Yoshitaka Nagai Kinya Ishikawa Gota Kawai Kazuhiko Nakatani
author_facet	Tomonori Shibata Konami Nagano Morio Ueyama Kensuke Ninomiya Tetsuro Hirose Yoshitaka Nagai Kinya Ishikawa Gota Kawai Kazuhiko Nakatani
author_sort	Tomonori Shibata
title	Small molecule targeting r(UGGAA) n disrupts RNA foci and alleviates disease phenotype in Drosophila model
title_short	Small molecule targeting r(UGGAA) n disrupts RNA foci and alleviates disease phenotype in Drosophila model
title_full	Small molecule targeting r(UGGAA) n disrupts RNA foci and alleviates disease phenotype in Drosophila model
title_fullStr	Small molecule targeting r(UGGAA) n disrupts RNA foci and alleviates disease phenotype in Drosophila model
title_full_unstemmed	Small molecule targeting r(UGGAA) n disrupts RNA foci and alleviates disease phenotype in Drosophila model
title_sort	small molecule targeting r(uggaa) n disrupts rna foci and alleviates disease phenotype in drosophila model
publisher	Nature Portfolio
publishDate	2021
url	https://doaj.org/article/fb74152ab4824a57b44c57a7b829b312
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Small molecule targeting r(UGGAA) n disrupts RNA foci and alleviates disease phenotype in Drosophila model

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