The AAV2-CHM virus was delivered in normal sighted mice and zebrafish, and the CHM gene was also induced in pluripotent stem cells (iPSCs) from patients with choroideremia with no evident toxicity [49,50], thus encouraging in vivo subretinal injection of the native AAV2-hCHM gene, which was reported to be safe and efficacious (NCT01461213 and NCT02341807) [170] for up to 3.5 years after treatment, despite progressive degeneration in the control eyes [171,172]. The gene discussed is CHM; the disease is choroideremia.