As a consequence of these multiple biologic effects, resulting in TP53 activation and MDM2 downregulation, in combination with transcriptional silencing of leukemia oncogenes, such as MYC, MYB and MCL-1, resulted in a rapid and pronounced anti-leukemia effects, confirmed in patient-pronounced anti-leukemia effects, confirmed in patient-derived xenografts of various AML subtypes, including TET-2−/− and FLT3-ITD driven leukemias [134]. The gene discussed is MDM2; the disease is leukemia.