New mRNA Delivery Platform Restores Muscle Function in DMD (Duchenne Muscular Dystrophy) Models
Mice with DMD (Duchenne Muscular Dystrophy) regain muscle function after targeted mRNA treatment: Engineered extracellular vesicles delivered full-length dystrophin mRNA to skeletal muscle of mice with Duchenne Muscular Dystrophy (DMD), restoring dystrophin production and improving muscle strength and function, according to a study in Nature Biomedical Engineering. Safety testing with non-human primates found the treatment was well tolerated, supporting its potential as a non-viral alternative to current gene therapies.