ABSTRACT

Gene therapy vectors based on adeno-associated virus (AAV) have garnered much attention for the potential treatment of Duchenne muscular dystrophy (DMD). These vectors are attractive due to their production characteristics, demonstrated muscle tropism, long lasting expression, and relatively low, but not absent, immunogenicity. In this chapter, we introduce some basic information on AAV and its recombinant vector (rAAV) derivatives and discuss their potential applications to gene therapy for DMD.