The RPE65 gene is one of the causative genes for LCA that has received the most scrutiny due to the approval of first‐in‐class gene therapy using a recombinant adeno‐associated virus (AAV) (voretigene neparvovec [VN]; Luxturna, Spark Therapeutics) (Russell et al. 2017). The gene discussed is RPE65; the disease is Leber congenital amaurosis.