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
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Acceso en línea:https://doaj.org/article/7ed353e7a01c44dbbbad10a4313b9a34
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