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...
Enregistré dans:
| Auteurs principaux: | , , |
|---|---|
| Format: | article |
| Langue: | EN |
| Publié: |
Nature Portfolio
2016
|
| Sujets: | |
| Accès en ligne: | https://doaj.org/article/7ed353e7a01c44dbbbad10a4313b9a34 |
| Tags: |
Ajouter un tag
Pas de tags, Soyez le premier à ajouter un tag!
|