Systemic AAV Gene Therapy Close to Clinical Trials for Several Neuromuscular Diseases

Abstract excerpt

While there have been a number of gene therapy clinical trials using adeno-associated viral (AAV) vectors, the majority have been safety studies involving local intramuscular delivery. To date, only one trial has involved systemic delivery: AAV-SMN1 for spinal muscular atrophy type 1 (Table 1). Likewise, many of the studies in large animals have focused on local or regional delivery (e.g., Qiao et al.1 and Le...

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