Viral vector-mediated gene therapies

Curr Opin Neurol. 2015 Oct;28(5):522-7. doi: 10.1097/WCO.0000000000000241.


Purpose of review: 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.

Recent findings: 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.

Summary: 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.

Publication types

  • Research Support, N.I.H., Extramural
  • Review

MeSH terms

  • Dependovirus / genetics*
  • Genetic Therapy / methods*
  • Genetic Vectors / therapeutic use*
  • Humans
  • Muscular Dystrophy, Duchenne / therapy*