In 2022, Zhang et al. published the first study of organ-specific gene augmentation therapy for the treatment of FD (Table 1), in which recombinant adeno-associated virus (rAAV) vector-mediated over-expression of ACDase ameliorated retinopathy in Asah1P361R/P361R mice [135]. The gene discussed is ASAH1; the disease is Fabry disease.