Article

A NEW DYSTROPHIN DEFICIENT RAT MODEL MIRRORING EXON SKIPPING IN PATIENTS WITH DMD EXON 45 DELETIONS

2025-06-02

Abstract excerpt

<h4>ABSTRACT</h4> Mutations in the dystrophin (DMD) gene can cause a spectrum of muscle-wasting disorders ranging from the milder Becker muscular dystrophy (BMD) to the more severe Duchenne muscular dystrophy (DMD). Among these, exon 45 deletion is the most frequently reported single exon deletion in DMD patients worldwide. In this study, we generated a novel rat model with an exon 45 deletion using CRISPR/Cas9 t...

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Literature Corpus work: 74ec5181-5acb-503b-9c9f-92732bd8a2fb
DOI: 10.1101/2025.05.30.656540

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