Based on our results, as well as work of other teams using murine and canine models of the disease39–43, Phase I/IIa clinical trials have been approved in human ACHM patients (NCT02935517 and NCT02610582), demonstrating the translational relevance of our work with this large animal model of CNGA3 ACHM . The gene discussed is CNGA3; the disease is achromatopsia.