The first in vivo viral gene therapy approved by the FDA for any indication in the United States was Voretigene neparvovec (Luxturna). It is an AAV2 vector encoding the normal form of retinal pigmented epithelium-65 (RPE-65) to counteract Leber congenital amaurosis, a rare form of inherited retinal photoreceptor disorder. This form of retinitis pigmentosa is an autosomal recessive disorder caused by mutation in both RPE-65 alleles. Patients present with diminished vision, typically from a very early age, that inexorably progresses and results in severely impaired vision or total blindness. Injection of AAV2 vectors encoding normal RPE65 significantly reduces that rate of visual loss and preserves retinal ganglion function for at least 7 years in humans, the longest time periods published to date. Treatment delivered earlier in life, when retinal photoreceptors are less damaged, appears to lead to better long-term outcomes. However, as data from longer timepoints post-treatment become available, concerns of potential late-developing adverse effects have been raised, including elevated intraocular pressure, cataracts, and chorioretinal atrophy. The approval of Voretigene neparvovec represented a major therapeutic advance for an otherwise untreatable disorder and a critical milestone in the advancement of gene therapies for human disorders. Gene therapy is currently under active study in other forms of retinitis pigmentosa and other inherited ocular disorders.