Some examples of disease indications in clinical trials are central nervous system disorders such as Parkinson’s disease (AAV2-GAD, AAV2-neurturin, AAV2-AADC), spinal muscular atrophy (AAV9-SMN), and lysosomal storage disorders such as MPS VII (AAV-GUSβ) [33]. The gene discussed is SMN1; the disease is proximal spinal muscular atrophy.