RPE65 and retinitis pigmentosa 1: Furthermore, understanding that the rod retinoid pathway is disrupted in these retinal diseases has also led to pharmacologic treatment options [34,35] shown in clinical trials to benefit not only patients with RP and LCA that possess a defective Rpe65 gene, but also patients that possess a defective Lrat (Lecithin retinol acyltransferase) gene [24].