Presently, three AAV-vector based gene therapeutics are available as Glybera, treating Lipoprotein Lipase (LPL)-deficiency, Luxturna [28], treating Retinal Pigment Epithelial (RPE) 65-related retinal dystrophy, and Zolgensma, treating Spinal Muscular Atrophy (SMA) [28,29,30]. The gene discussed is LPL; the disease is proximal spinal muscular atrophy.