To date, there are three FDA approved gene therapies, including Spinraza (nusinersen; anti-sense oligonucleotide for treatment of spinal muscular atrophy type 1) (64, 65), Kymriah (tisagenlecleucel; CAR T-cell therapy for treatment of B-cell precursor acute lymphoblastic leukemia) (66), and Luxturna (voretigene neparvovec; adeno-associated virus-mediated delivery of RPE65 for treatment of RPE65-mediated inherited retinal dystrophy) (67). Here, RPE65 is linked to spinal muscular atrophy, type 1.