adeno-associated virus vector micro-dystrophin

Adeno-associated virus (AAV) vector micro-dystrophin is a gene therapy approach designed to treat Duchenne muscular dystrophy (DMD). This modality utilizes a recombinant AAV vector (such as AAVrh74 or AAV9) to deliver a miniaturized, functional version of the human dystrophin gene, known as micro-dystrophin, to skeletal and cardiac muscle cells. The full-length dystrophin gene is too large for AAV packaging, necessitating the use of these truncated variants that retain essential domains. Expression is typically controlled by muscle-specific promoters to ensure targeted activity. The resulting micro-dystrophin protein localizes to the muscle cell membrane (sarcolemma), where it stabilizes the dystrophin-associated protein complex (DAPC). This stabilization protects muscle fibers from mechanical stress during contraction, thereby reducing necrosis, fibrosis, and the progressive muscle weakness characteristic of DMD.

Other names

AAV-micro-dystrophinAAV-μDysmicro-dystrophin gene therapyrAAV-micro-dystrophin