Question

Excision of expanded gaa repeats alleviates the molecular phenotype of friedreich's ataxia

Answer 1

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.