Gene therapy for Duchenne muscular dystrophy: AAV leads the way.

Over the past decade, adeno-associated virus (AAV) has become an extremely promising vector for gene therapy of many genetic disorders. This review summarizes the specific challenges that must be overcome to apply AAV gene therapy to Duchenne muscular dystrophy. Many of these challenges have been met successfully in animal studies, but further work is needed to translate these results into an effective clinical treatment.