Several phase I/II clinical trials for gene therapy using AAV to deliver CNGA3 and CNGB3 are currently in process for the treatment of ACHM [63].Genome editing in hiPSCs represents an enormous tool for disease investigation and molecular and cellular research avoiding the use of viral vectors to introduce exogenous material, as occurs with other therapeutic approximations.Additionally, gene editing enables the permanent correction of pathogenic variants in a patient’s hiPSCs, which are a potentially unlimited cellular source for autologous cellular therapy. Here, CNGA3 is linked to achromatopsia.