CHM and choroideremia: Preclinical proof-of-concept studies on the feasibility of CHM gene replacement have been conducted both in vitro, by inducing the CHM gene in pluripotent stem cells (iPSCs) from patients with CHM, and in vivo, by delivering the AAV2-CHM virus in normal sighted mice and zebrafish, with no evidence of toxicity (215, 216), paving the way to clinical trials on CHM patients.