dystrophin gene therapy (AAV-mediated)

AAV-mediated dystrophin gene therapy refers to a class of genetic therapies designed to treat Duchenne muscular dystrophy (DMD) by delivering a functional, truncated version of the dystrophin gene (such as micro-dystrophin or mini-dystrophin) using adeno-associated virus (AAV) vectors. Because the full-length dystrophin gene is too large to fit within the packaging capacity of standard AAV vectors, these therapies utilize engineered, shortened constructs that retain essential functional domains of the dystrophin protein. Delivered systemically, the therapy aims to restore dystrophin expression in skeletal and cardiac muscle tissues, thereby stabilizing the sarcolemma, reducing muscle degeneration, and slowing the progression of muscle wasting and associated cardiomyopathy.

Other names

AAV-mediated dystrophin gene therapyAAV-mediated micro-dystrophin gene therapyAAV-mediated mini-dystrophin gene therapydystrophin gene therapymicro-dystrophin gene therapymini-dystrophin gene therapy