Activating frataxin expression by repeat-targeted nucleic acids

Liande Li, Masayuki Matsui & David R. Corey; Nature Communications 7, Article number: 10606 doi:10.1038/ncomms10606

Iintroducing anti-GAA duplex RNAs or single-stranded locked nucleic acids into patient-derived cells increases FXN protein expression to levels similar to analogous wild-type cells. Synthetic nucleic acids that target GAA repeats can be lead compounds for restoring curative FXN levels.