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Networking to Optimize Dmd exon 53 Skipping in the Brain of mdx52 Mouse Model

7 Dec 2023

Abstract excerpt

Duchenne muscular dystrophy (DMD) is caused by mutations in the DMD gene that disrupt the open reading frame and thus prevent production of functional dystrophin proteins. Recent advances in DMD treatment, notably exon skipping and AAV gene therapy, have achieved some success aimed at alleviating the symptoms related to progressive muscle damage. However, they do not address the brain comorbidities associated...

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PMID: 38137463
DOI: 10.3390/biomedicines11123243

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