Micro-dystrophin protein (micro-dystrophin)

Micro-dystrophin protein is a synthetic, truncated form of dystrophin engineered to retain essential functional domains and fit the limited packaging capacity of adeno-associated virus (AAV) vectors used in gene therapy[2][3][6][7]. Unlike full-length dystrophin, micro-dystrophin lacks significant sections of the rod domain and distal C-terminus but preserves actin-binding and β-dystroglycan-binding regions critical for muscle cell membrane stability[1][2]. Overexpression via AAV-mediated gene transfer is a promising treatment for Duchenne muscular dystrophy (DMD), a genetic disorder resulting from mutations in the DMD gene and absence of functional dystrophin[7][8]. Clinical trials focus on delivery efficacy, expression levels, immune responses, and long-term therapeutic benefit, but micro-dystrophin is not a receptor or enzyme, rather it is a therapeutic replacement protein[1][2][5][6].

Other names

Mini-dystrophintruncated dystrophinsynthetic micro-dystrophin

Replacement therapy: Expression of micro-dystrophin in muscle cells via gene transfer restores some structural/functional capacity lost in Duchenne muscular dystrophy[7][8].