A new preclinical and translational package reports systemic delivery of full-length Duchenne muscular dystrophy mRNA using skeletal-muscle-targeted extracellular vesicles (EVs). In the study, researchers used engineered EVs (DMD t-EVs) that deliver full-length dystrophin mRNA and restore dystrophin production in murine DMD models, with reported improvements in muscle strength and endurance. The work also reported safety and biocompatibility signals in non-human primates, addressing a key hurdle for non-viral gene delivery: tolerability and delivery efficiency at scale. The study positions EV-based mRNA as a way to bypass vector limitations that restrict full-length DMD gene transfer. If subsequent studies validate efficacy durability and immune response profiles, this approach could broaden the competitive landscape beyond viral gene therapy formats in DMD.
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