Viral vector-mediated gene therapies

Gene therapy as a treatment for neuromuscular disease has significantly advanced over the past decade. In the present review, the progress of adeno-associated viruses (AAV) vector-mediated gene therapy for Duchenne muscular dystrophy (DMD) during the past year is highlighted.

Modulating the immune response to AAV vector capsid or the transgene has helped to increase stable transduction efficiency. Full-length dystrophin expression via gene editing with targeted nucleases may ultimately be an ideal treatment option. Also genes with homologues function may ameliorate many aspects of the DMD pathophysiology.

The work during the past year has increased our understanding of AAV vector-mediated therapy and has also validated new approaches to treat DMD. The results will aid in the design of both preclinical and clinical trials.