Article

Validation of Duchenne muscular dystrophy candidate modifiers using a CRISPR-Cas9-based approach in zebrafish

2025-05-20

Abstract excerpt

Duchenne muscular dystrophy (DMD) is a progressive muscle wasting disease for which there is no cure. There is a critical need for additional therapeutics. Human genome-wide association studies (GWAS) have identified candidate DMD genetic modifiers that could serve as therapeutic targets. Because many GWAS-identified single nucleotide polymorphisms (SNPs) lie in noncoding, putative regulatory regions, it can be ch...

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Literature Corpus work: eb8b5697-5ae4-5e5d-bf9a-662ca387b91f
DOI: 10.1101/2025.05.20.655139

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