RPE65 and inherited retinal dystrophy: While AD remains a daunting biological and clinical problem, recently, a number of other devastating diseases long thought to be intractable—including spinal muscular atrophy (Al-Zaidy et al. 2019), transfusion-dependent β-thalassemia (Thompson et al. 2018), hemophilia A (Garde 2019), and biallelic RPE65-mediated inherited retinal disease (Voretigene neparvovec-rzyl (Luxturna) for inherited retinal dystrophy 2018)—have begun to be treated with somatic-cell and gene therapies (albeit very expensive ones—more on that below).