RPE65 and choroideremia: Current work therefore builds on design principles, safety data, and efficacy standards established by mature IRD gene therapies, including gene augmentation therapy LUXTURNA for RPE65-associated LCA2, CRISPR/Cas9 therapy for CEP290-associated LCA10, AAV-mediated therapy for choroideremia (CHM), and phase I/II/III trials for XLRP.