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Duchenne muscular dystrophy (DMD) is the most common lethal muscle disorder of children.It is caused by mutations of the dystrophin gene.Adeno-associated virus (AAV)-mediated gene replacement therapy has been actively pursued for DMD therapy in last 12 years.Due to the inherent limitation of the AAV packaging capacity, the main focus of the field has been on delivering synthetic mini-and micro-dystrophin genes.