This study explores direct reprogramming of DMD fibroblasts into myotubes as a non-invasive model for testing antisense oligonucleotide (AO)-mediated exon skipping. Using MyoD gene induction, researchers converted fibroblasts into muscle cells, then applied phosphorodiamidate morpholino oligomers (PMOs) to skip exons 45–55, restoring dystrophin expression. This scalable, reproducible method provides an alternative to muscle biopsies, advancing DMD drug development.

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Multiple exon skipping is a promising therapy for Duchenne Muscular Dystrophy (DMD), targeting genetic hot spots like exons 45–55 and 3–9 to restore dystrophin. While PMOs have shown potential, challenges in delivery remain. Advances like vivo-PMOs aim to enhance uptake. Despite promising preclinical results, clinical validation is needed to ensure effectiveness and long-term safety. Ongoing research focuses on optimizing delivery and broadening patient eligibility for exon-skipping therapies.

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