rAAV1-CMV-miniDMD

rAAV1-CMV-miniDMD is an experimental gene therapy vector consisting of a recombinant adeno-associated virus serotype 1 (rAAV1) encoding a miniaturized form of the dystrophin gene (miniDMD) under the control of the cytomegalovirus (CMV) promoter. The therapy is designed to deliver the mini-dystrophin transgene to muscle tissue, enabling expression of a functional but truncated dystrophin protein in cells affected by Duchenne muscular dystrophy (DMD). The CMV promoter allows efficient transduction of striated muscle, and the reduced gene size accommodates the packaging limits of AAV vectors. Preclinical studies demonstrated efficient long-term transgene expression of the mini-dystrophin in the muscular component of the diaphragm and peritoneum in rhesus monkeys, with no adverse effects observed[1][3]. The approach offers potential for early, durable gene replacement therapy in genetic muscle disorders.

Other names

rAAV1-CMV-miniDMDrAAV-1-CMV-miniDMDrAAV 1-CMV-miniDMD