rAAVrh74.DV.minidystrophin

rAAVrh74.DV.minidystrophin is an experimental gene therapy construct designed for the treatment of Duchenne muscular dystrophy (DMD). It utilizes a recombinant adeno-associated virus serotype rh74 (rAAVrh74) vector to deliver a "mini-dystrophin" transgene. This specific construct is distinguished by its inclusion of the neuronal nitric oxide synthase (nNOS) binding site (typically spectrin repeats R16 and R17), which is often omitted in smaller "micro-dystrophin" versions. The inclusion of the nNOS binding site is intended to localize nNOS to the sarcolemma, potentially improving muscle blood flow and reducing exercise-induced fatigue. Developed through research at Nationwide Children's Hospital in collaboration with Sarepta Therapeutics, this construct was evaluated in preclinical studies using the mdx mouse model to compare its histological and functional efficacy against various micro-dystrophin designs, including those using the MHCK7 and MCK promoters.

Other names

rAAVrh74-DV-minidystrophinrAAVrh-74-DV-minidystrophinrAAVrh 74-DV-minidystrophinmini-dystrophin (nNOS) gene therapy