RPE65 and proximal spinal muscular atrophy: The treatment of familial lipoprotein lipase deficiency (LPLD) with Glybera (a rAAV1 encoding the lipoprotein lipase variant LPLS447X; Ylä-Herttuala, 2012), a rare form of inherited blindness with Luxturna (a rAAV2 encoding a normal copy of the retinal pigment epithelium-specific 65 kDa protein; Russell et al., 2017), and spinal muscular atrophy (SMA) with Zolgensma (a rAAV9 encoding the survival motor neuron 1 protein; Hoy, 2019) provide examples of approved products.