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...
| Main Authors: | , , |
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| Format: | Article |
| Language: | English |
| Published: |
Nature Publishing Group
2016-02-01
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| Series: | Nature Communications |
| Online Access: | https://doi.org/10.1038/ncomms10606 |