The current mainstay for the treatment of w-AMD and DME is represented by intravitreal drugs targeting the vascular endothelial growth factor (VEGF), which plays a crucial role in the pathogenesis of these exudative macular disorders [5]; however, real-life studies with anti-VEGF agents for treating w-AMD and DME have frequently shown inferior results as compared with pivotal clinical trials, due to the need of frequent injections, challenges with optimal disease monitoring, high costs and suboptimal response in some patients [6,7,8]. The gene discussed is VEGFA; the disease is age-related macular degeneration.