rAAV2.5-CMV-minidystrophin

rAAV2.5-CMV-minidystrophin is an investigational gene therapy designed for the treatment of Duchenne muscular dystrophy (DMD). It uses a recombinant adeno-associated virus serotype 2.5 (rAAV2.5) vector to deliver a miniaturized, functional version of the human dystrophin gene (minidystrophin) under the control of a cytomegalovirus (CMV) promoter into muscle cells[1][7]. The goal is to restore production of dystrophin protein in patients with DMD, whose native gene is mutated and nonfunctional, thereby improving muscle function and slowing disease progression[1][6]. Clinical trials have shown that this approach can lead to expression of minidystrophin in muscle fibers; however, immune responses against the newly synthesized protein have limited its effectiveness in some patients[1][7]. The therapy was developed by Asklepion Pharmaceuticals and has been tested in early-phase clinical trials for safety and preliminary efficacy in boys with DMD[5].

Other names

Muscular dystrophy gene therapy