While gene therapy is currently being tested only for terminal diseases like haemophilia and AIDS, this innovation has shown promising progress during the past two decades, with a handful of notable successes including treatments for the X-linked severe combined immunodeficiency and the inherited blindness Leber’s congenital amaurosis 2, which are caused by mutations in the interleukin-2 receptor γ chain (IL2RG) and the retinal pigment epithelium-specific 65 kDa protein (RPE65) genes, respectively [99,100]. Here, RPE65 is linked to severe combined immunodeficiency.