Excision of expanded gaa repeats alleviates the molecular phenotype of friedreich's ataxia
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Friedreich's ataxia (FRDA) is an autosomal recessive neurological disease caused by expansions of guanine-adenine-adenine (GAA) repeats in intron 1 of the frataxin (FXN) gene. ... We report that human FRDA cells can be corrected by zinc finger nuclease-mediated excision of the expanded GAA repeats.
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