Integrating vector and stem cell-based strategies for gene therapy of Duchenne muscular dystrophy

We review novel gene transfer strategies proposed to be suitable for the treatment Duchenne Muscular Dystrophy (DMD): use of hybrid adeno-retroviral vectors to stably replace dystrophin ultimately in patients lacking this gene and the potential intravenous application of stem cells and monocytes for targeted gene transfer. We discuss the limitations of current vector technology and demonstrate the need for continual evolution of vector design that is required before gene therapy of complex monogenic diseases such as DMD becomes a reality.