Article

Deletion of exons 45 to 55 in the <i>DMD</i> gene: from the therapeutic perspective to the <i>in vitro</i> model

2023-09-14

Abstract excerpt

<h4>ABSTRACT</h4> Gene editing therapies in development for correcting out-of-frame DMD mutations in Duchenne muscular dystrophy aim to replicate benign spontaneous deletions. Deletion of 45 – 55 DMD exons (del45 – 55) was described in asymptomatic subjects, but recently serious skeletal and cardiac complications have been reported. Uncovering why a single mutation like del45–55 is able to induce diverse phen...

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Literature Corpus work: 3ebeecb8-200d-522f-bf12-20695e79b319
DOI: 10.1101/2023.09.13.557649

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