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Activating frataxin expression by repeat-targeted nucleic acids

Friedreich's ataxia is a genetic disorder caused by a mutant expansion of the trinucleotide GAA within an intronic FXN RNA. This expansion leads to reduced expression of frataxin (FXN) protein and evidence suggests that transcriptional repression is caused by a specific structure, called and R-loop, that forms between the expanded repeat RNA and complementary genomic DNA. Synthetic agents that increase levels of FXN protein might alleviate the disease. We demonstrate that introducing specific oligonucleotide molecules into patient-derived cells increases FXN protein expression to levels similar to that in cells from unaffected individuals. Our data are significant because synthetic nucleic acids that target GAA repeats can be lead compounds for restoring curative FXN levels.

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