Article

Quercetin selectively reduces expanded repeat RNA levels in models of myotonic dystrophy

2023-02-02

Abstract excerpt

<h4>ABSTRACT</h4> Myotonic dystrophy is a multisystemic neuromuscular disease caused by either a CTG repeat expansion in DMPK (DM1) or a CCTG repeat expansion in CNBP (DM2). Transcription of the expanded alleles produces toxic gain-of-function RNA that sequester the MBNL family of alternative splicing regulators into ribonuclear foci, leading to pathogenic mis-splicing. There are currently no approved treatment...

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Literature Corpus work: 0e2e202f-a7a5-59e4-b30c-35951cc59079
DOI: 10.1101/2023.02.02.526846

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