Article

Treating Myotonic Dystrophy with artificial RNA endonucleases to specifically degrade toxic RNA expansions

2025-06-13

Abstract excerpt

<h4>ABSTRACT</h4> Myotonic dystrophy type 1 (DM1), the most common autosomal dominant muscular disorder, is driven by expanded CUG repeats in the 3′ UTR of DMPK gene, which sequester RNA-binding proteins and cause aberrant alternative splicing. Despite extensive study, no effective treatment exists, and current care remains limited to symptomatic management. Using a customized dual-color reporter carrying RNA rep...

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Literature Corpus work: 51425b83-a14f-5b7c-b22c-65acb352b0bf
DOI: 10.1101/2025.06.10.658795

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