In AD, a nilotinib (c-Abl orthosteric inhibitor) trial of 37 patients using the maximal daily tolerated dose of 300 mg showed that after 6–12 months of treatment, the group had reduced Aβ40 and Aβ42 cerebrospinal fluid levels compared to the placebo group, displaying also reduced Tau phosphorylation (Turner et al., 2020). The gene discussed is MAPT; the disease is Alzheimer disease.