dysferlin mRNA-loaded extracellular vesicles

Dysferlin mRNA-loaded extracellular vesicles are engineered extracellular vesicles (EVs) designed to deliver full-length dysferlin transcripts to dysferlin-deficient muscle cells [SP47]. Developed by researchers at The Ohio State University, these muscle-lineage EVs are generated through the transient overexpression of dysferlin in donor myoblasts [SP47]. By delivering dysferlin mRNA alongside other cytoskeletal and sarcolemmal repair-related proteins, these engineered EVs aim to restore calcium-dependent plasma membrane repair and calcium handling in patient-derived myoblasts [SP47]. This non-viral nanocarrier platform represents a promising therapeutic strategy for dysferlinopathies, such as limb-girdle muscular dystrophy type 2B (LGMD2B/R2) and Miyoshi myopathy, which are progressive degenerative muscle disorders caused by mutations in the dysferlin gene [SP47].