Activating frataxin expression by repeat-targeted nucleic acids

Expansion of the trinucleotide GAA within an intronic FXN RNA can cause Friedreich's Ataxia (FRDA), an incurable genetic disorder. Here, the authors show that anti-GAA duplex RNAs or single-stranded locked nucleic acids increases FXN protein expression in patient-derived cells to levels similar...

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Autores principales:	Liande Li, Masayuki Matsui, David R. Corey
Formato:	article
Lenguaje:	EN
Publicado:	Nature Portfolio 2016
Materias:	Science Q
Acceso en línea:	https://doaj.org/article/7ed353e7a01c44dbbbad10a4313b9a34
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id	oai:doaj.org-article:7ed353e7a01c44dbbbad10a4313b9a34
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spelling	oai:doaj.org-article:7ed353e7a01c44dbbbad10a4313b9a342021-12-02T16:49:26ZActivating frataxin expression by repeat-targeted nucleic acids10.1038/ncomms106062041-1723https://doaj.org/article/7ed353e7a01c44dbbbad10a4313b9a342016-02-01T00:00:00Zhttps://doi.org/10.1038/ncomms10606https://doaj.org/toc/2041-1723Expansion of the trinucleotide GAA within an intronic FXN RNA can cause Friedreich's Ataxia (FRDA), an incurable genetic disorder. Here, the authors show that anti-GAA duplex RNAs or single-stranded locked nucleic acids increases FXN protein expression in patient-derived cells to levels similar to wild-type cells.Liande LiMasayuki MatsuiDavid R. CoreyNature PortfolioarticleScienceQENNature Communications, Vol 7, Iss 1, Pp 1-8 (2016)
institution	DOAJ
collection	DOAJ
language	EN
topic	Science Q
spellingShingle	Science Q Liande Li Masayuki Matsui David R. Corey Activating frataxin expression by repeat-targeted nucleic acids
description	Expansion of the trinucleotide GAA within an intronic FXN RNA can cause Friedreich's Ataxia (FRDA), an incurable genetic disorder. Here, the authors show that anti-GAA duplex RNAs or single-stranded locked nucleic acids increases FXN protein expression in patient-derived cells to levels similar to wild-type cells.
format	article
author	Liande Li Masayuki Matsui David R. Corey
author_facet	Liande Li Masayuki Matsui David R. Corey
author_sort	Liande Li
title	Activating frataxin expression by repeat-targeted nucleic acids
title_short	Activating frataxin expression by repeat-targeted nucleic acids
title_full	Activating frataxin expression by repeat-targeted nucleic acids
title_fullStr	Activating frataxin expression by repeat-targeted nucleic acids
title_full_unstemmed	Activating frataxin expression by repeat-targeted nucleic acids
title_sort	activating frataxin expression by repeat-targeted nucleic acids
publisher	Nature Portfolio
publishDate	2016
url	https://doaj.org/article/7ed353e7a01c44dbbbad10a4313b9a34
work_keys_str_mv	AT liandeli activatingfrataxinexpressionbyrepeattargetednucleicacids AT masayukimatsui activatingfrataxinexpressionbyrepeattargetednucleicacids AT davidrcorey activatingfrataxinexpressionbyrepeattargetednucleicacids
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Activating frataxin expression by repeat-targeted nucleic acids

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