mini-dystrophin

Mini-dystrophin is a genetically engineered, truncated version of the full-length dystrophin protein designed for use in gene therapy for Duchenne muscular dystrophy (DMD). The full-length dystrophin gene is too large to be packaged into commonly used viral vectors such as adeno-associated virus (AAV), so researchers have developed smaller versions—mini- and micro-dystrophins—that retain key functional domains necessary for stabilizing muscle cell membranes and linking the cytoskeleton to the extracellular matrix[1][7]. Mini-dystrophins are based on naturally occurring deletions found in some Becker muscular dystrophy patients with milder symptoms, and they are designed to restore partial function lost in DMD[1][6]. These constructs can be delivered via AAV vectors directly into muscle cells, where they express a functional protein that helps protect muscle fibers from damage during contraction[7]. However, early clinical trials have shown challenges such as immune responses against the newly synthesized protein and variable efficacy[5][6].

Other names

minidystrophinrAAV2.5-CMV-minidystrophinrAAV-2.5-CMV-minidystrophinrAAV 2.5-CMV-minidystrophin