microdystrophin

**Microdystrophin** is a genetically engineered, truncated form of the dystrophin protein designed to maintain essential domains for muscle function and stability but small enough to be packaged and delivered using adeno-associated virus (AAV) vectors, which are limited in genetic cargo capacity. The development of microdystrophin addresses the inability to deliver the full-length dystrophin gene, which is too large for AAV vectors. Microdystrophins retain the critical elements necessary for dystrophin's role in linking the subsarcolemmal cytoskeleton and the dystrophin-associated protein complex (DAPC), thereby stabilizing muscle fibers and reducing muscle damage in Duchenne muscular dystrophy (DMD). Microdystrophin gene therapy is primarily developed as a treatment for DMD, an X-linked neuromuscular disorder caused by mutations in the dystrophin gene. Multiple vectors containing microdystrophin variants are being studied, most notably the therapy marketed as delandistrogene moxeparvovec (Elevidys), which uses AAVrh74 to deliver the microdystrophin transgene specifically to skeletal and cardiac muscle cells[1][2][3][5][6].

Other names

micro-dystrophinmicro dystrophin