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...

Full description

Saved in:
Bibliographic Details
Main Authors: Liande Li, Masayuki Matsui, David R. Corey
Format: article
Language:EN
Published: Nature Portfolio 2016
Subjects:
Q
Online Access:https://doaj.org/article/7ed353e7a01c44dbbbad10a4313b9a34
Tags: Add Tag
No Tags, Be the first to tag this record!