DMD and Duchenne muscular dystrophy: rAAV2.5 mini-dystrophin was delivered through intramuscular injection in the bicep muscle of six DMD boys (Phase I clinical trial, NCT00428935) which lead to effective gene delivery to the muscle cells with low immunological reaction to the viral vector; nevertheless, only very few dystrophin-positive myofibres were detected in two of the six patients [115,116].