Short antisense oligonucleotides alleviate the pleiotropic toxicity of RNA harboring expanded CGG repeats

Fragile X-associated tremor/ataxia syndrome is a neurodegenerative disease caused by toxic RNA containing expanded CGG repeats. Here, the authors show that synthetic oligonucleotides targeting the RNA repeats decrease the pleiotropic effect of this toxic molecule in cellular and animal models of the...

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Autores principales:	Magdalena Derbis, Emre Kul, Daria Niewiadomska, Michał Sekrecki, Agnieszka Piasecka, Katarzyna Taylor, Renate K. Hukema, Oliver Stork, Krzysztof Sobczak
Formato:	article
Lenguaje:	EN
Publicado:	Nature Portfolio 2021
Materias:	Science Q
Acceso en línea:	https://doaj.org/article/c6498e46fb154abdbc4b08a40653c26d
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spelling	oai:doaj.org-article:c6498e46fb154abdbc4b08a40653c26d2021-12-02T15:53:25ZShort antisense oligonucleotides alleviate the pleiotropic toxicity of RNA harboring expanded CGG repeats10.1038/s41467-021-21021-w2041-1723https://doaj.org/article/c6498e46fb154abdbc4b08a40653c26d2021-02-01T00:00:00Zhttps://doi.org/10.1038/s41467-021-21021-whttps://doaj.org/toc/2041-1723Fragile X-associated tremor/ataxia syndrome is a neurodegenerative disease caused by toxic RNA containing expanded CGG repeats. Here, the authors show that synthetic oligonucleotides targeting the RNA repeats decrease the pleiotropic effect of this toxic molecule in cellular and animal models of the disease.Magdalena DerbisEmre KulDaria NiewiadomskaMichał SekreckiAgnieszka PiaseckaKatarzyna TaylorRenate K. HukemaOliver StorkKrzysztof SobczakNature PortfolioarticleScienceQENNature Communications, Vol 12, Iss 1, Pp 1-17 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	Science Q
spellingShingle	Science Q Magdalena Derbis Emre Kul Daria Niewiadomska Michał Sekrecki Agnieszka Piasecka Katarzyna Taylor Renate K. Hukema Oliver Stork Krzysztof Sobczak Short antisense oligonucleotides alleviate the pleiotropic toxicity of RNA harboring expanded CGG repeats
description	Fragile X-associated tremor/ataxia syndrome is a neurodegenerative disease caused by toxic RNA containing expanded CGG repeats. Here, the authors show that synthetic oligonucleotides targeting the RNA repeats decrease the pleiotropic effect of this toxic molecule in cellular and animal models of the disease.
format	article
author	Magdalena Derbis Emre Kul Daria Niewiadomska Michał Sekrecki Agnieszka Piasecka Katarzyna Taylor Renate K. Hukema Oliver Stork Krzysztof Sobczak
author_facet	Magdalena Derbis Emre Kul Daria Niewiadomska Michał Sekrecki Agnieszka Piasecka Katarzyna Taylor Renate K. Hukema Oliver Stork Krzysztof Sobczak
author_sort	Magdalena Derbis
title	Short antisense oligonucleotides alleviate the pleiotropic toxicity of RNA harboring expanded CGG repeats
title_short	Short antisense oligonucleotides alleviate the pleiotropic toxicity of RNA harboring expanded CGG repeats
title_full	Short antisense oligonucleotides alleviate the pleiotropic toxicity of RNA harboring expanded CGG repeats
title_fullStr	Short antisense oligonucleotides alleviate the pleiotropic toxicity of RNA harboring expanded CGG repeats
title_full_unstemmed	Short antisense oligonucleotides alleviate the pleiotropic toxicity of RNA harboring expanded CGG repeats
title_sort	short antisense oligonucleotides alleviate the pleiotropic toxicity of rna harboring expanded cgg repeats
publisher	Nature Portfolio
publishDate	2021
url	https://doaj.org/article/c6498e46fb154abdbc4b08a40653c26d
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Short antisense oligonucleotides alleviate the pleiotropic toxicity of RNA harboring expanded CGG repeats

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