Article

Anti-gene oligonucleotides targeting Friedreich’s ataxia expanded GAA•TTC repeats increase Frataxin expression

2024-01-25

Abstract excerpt

<h4>ABSTRACT</h4> Friedreich’s ataxia (FRDA) is a progressive, autosomal recessive ataxia caused, in the majority of cases, by homozygous expansion of GAA•TTC triplet-repeats in the first intron of the frataxin ( FXN ) gene. GAA•TTC repeat expansion results in the formation of non-B DNA intramolecular triplex structure (H-DNA) as well as changes in the epigenetic landscape at FXN loci and heterochromatin forma...

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Literature Corpus work: 8e0a4fee-f64d-5be5-8346-cbc243f1e361
DOI: 10.1101/2024.01.25.577034

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