AAV8 microdystrophin

AAV8 microdystrophin is an investigational gene therapy designed for the treatment of Duchenne muscular dystrophy (DMD). It utilizes an adeno-associated virus serotype 8 (AAV8) vector to deliver a codon-optimized microdystrophin transgene to muscle cells. This shortened dystrophin gene construct is engineered to restore dystrophin function in muscle (both skeletal and cardiac), aiming to stabilize muscle cell membranes during contraction, reduce muscle degeneration, and improve muscle function. The therapy is delivered as a one-time intravenous infusion, with clinical programs developed and run by companies such as Genethon and Regenxbio. Early clinical trials report sustained microdystrophin expression, reductions in muscle damage biomarkers, and stabilization or improvement in motor function over one to two years. The primary mechanism is gene replacement, with tissue-specific promoters enabling expression in muscle and heart[2][3][4][6][9][10][12][14].

Other names

AAV8-microdystrophinAAV-8-microdystrophinAAV 8-microdystrophinAAV8 micro-dystrophinAAV-8 micro-dystrophinAAV 8 micro-dystrophinAAV-microdystrophin gene therapy