Article

Large-scale expansions of Friedreich’s ataxia GAA•TTC repeats in human cells are prevented by LNA-DNA oligonucleotides and PNA oligomers

2022-07-05

Abstract excerpt

The human disease Friedreich’s ataxia (FRDA) is caused by expansions of GAA•TTC repeats in the first intron intron of the frataxin ( FXN) gene, and both intergenerational and somatic expansions are crucial for disease development. We and others have shown earlier that expanded GAA•TTC repeats can form an intramolecular triplex structure (H-DNA). Here we studied the effects of locked nucleic acid (LNA)-DNA mixmer...

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Literature Corpus work: ca4d4ebb-69ed-5749-a192-226c459d3308
DOI: 10.1101/2022.07.04.498742

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Large-scale expansions of Friedreich’s ataxia GAA•TTC repeats in human cells are prevented by LNA-DNA oligonucleotides and PNA oligomersDOI 10.1101/2022.07.04.498742Read

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