Article

Reproducible Computational Framework for Precision RNA Targeting in Myotonic Dystrophy Type 1: Balancing ASO Specificity and Cas13 Potency at the DMPK Locus

2026-01-06

Abstract excerpt

<title>Abstract</title> <p> Myotonic dystrophy type 1 (DM1) is caused by toxic CTG repeat expansions in the 3′UTR of the <italic>DMPK</italic> gene, leading to pathogenic RNA gain-of-function effects and widespread splicing abnormalities. RNA-targeting strategies such as antisense oligonucleotides (ASOs) and CRISPR-Cas13 hold strong therapeutic promise, but require reproducible design frameworks that balance s...

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Literature Corpus work: aa5e4f93-7d14-5a13-813d-c7b7225166d7
DOI: 10.21203/rs.3.rs-8456938/v1

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