Abstract
There is considerable interest, excitement and anticipation for the development of adeno associated virus (AAV) gene therapy as a treatment for Duchenne muscular dystrophy (DMD). Much of this expectation is based on promising preclinical data as well as success in other neuromuscular conditions, such as spinal muscular atrophy (SMA). However, it is also important to take an objective view of the realistic possibilities and limitations of AAV gene therapy for DMD as well as considering the likely barriers to clinical trials and the development of gene therapy as an approved and accessible treatment.
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