New Molecular Insights into Duchenne and Becker Muscular Dystrophy
01.10.2026
Our new study provides important insights into the molecular mechanisms underlying Duchenne (DMD) and Becker muscular dystrophy (BMD) using genetically tailored pig models. By analyzing gene expression and microRNA (miRNA) regulatory networks in skeletal muscle, we identified distinct molecular signatures associated with severe DMD and the milder BMD-like condition. Remarkably, restoring the dystrophin reading frame through exon 51 skipping partially normalized muscle-related gene expression and reduced disease-associated molecular changes. We also identified two promising miRNA biomarker candidates, miR-296-3p and miR-423-5p, that distinguish the two disease forms. These findings highlight the value of genetically modified pig models for understanding muscular dystrophies and advancing the development of RNA-based therapies and diagnostic biomarkers.