AAV2.5-minidystrophin

AAV2.5-minidystrophin is an investigational gene therapy designed to treat Duchenne muscular dystrophy (DMD) by delivering a truncated version of the human dystrophin gene (mini-dystrophin) using a recombinant adeno-associated virus vector, specifically the AAV2.5 serotype[1][3]. The mini-dystrophin transgene is typically expressed under the control of a muscle-specific or ubiquitous promoter such as CMV[1]. The goal is to restore partial function of dystrophin in muscle cells, thereby ameliorating disease pathology and improving muscle integrity and function[3]. Preclinical studies in animal models have shown that delivery of minidystrophins via AAV vectors can prevent muscle degeneration and restore normal histology in affected muscles[3].

Other names

mini-dystrophin gene therapyrecombinant adeno-associated virus carrying mini-dystrophin