PubMed · 2026-04-13
This review examines how tiny cell-secreted particles called extracellular vesicles (especially exosomes) could become new treatment tools for Duchenne muscular dystrophy, a severe muscle-wasting disease. These vesicles can carry gene-editing tools and healing molecules into muscle cells, potentially restoring function where current therapies fall short.
Current DMD treatments including antisense oligonucleotides, CRISPR/Cas9, and cell transplantation can delay but not cure the disease.
Natural extracellular vesicles enriched with regenerative microRNAs and anti-fibrotic proteins can modulate inflammation, oxidative stress, and muscle degeneration.
Engineered exosomes show potential as delivery vehicles for oligonucleotides and CRISPR/Cas9 components to restore dystrophin expression, though challenges in isolation, stability, and scalability remain.