Researchers at UT MD Anderson and collaborators published results in Nature Biomedical Engineering showing skeletal-muscle-targeted non-viral delivery of full-length DMD mRNA in a murine model. The approach uses allogenically engineered extracellular vesicles (DMD t-EVs) with targeting tags that direct cargo to skeletal muscle after systemic administration. The study reports restored dystrophin production alongside improvements in muscle strength, endurance, and overall function in vivo. It also includes preclinical safety and biocompatibility data from non-human primates, addressing one of the major translational hurdles for non-viral gene and RNA therapies. The work positions EV-based RNA delivery as a potential alternative to viral vectors, specifically aiming to overcome packaging limits for the longest known human gene while reducing immune and toxicity risks associated with viral approaches.
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