Inhibition of CDK2 effectively arrested tumor growth and prolonged the survival of AML-bearing mice. (A-F) Intratumoral therapy with the lentivirus encoding shRNA targeting CDK2 arrests tumor growth and induces human AML xenograft differentiation. (A) Images of mice and tumors in each group on day 10. (B) Tumor growth of HL60 xenografts. Tumor volume growth curves are expressed as the mean ± standard error, n = 5. Red arrow indicates the time of intratumoral injection. (C) Body weight of each group. (D) Effect of intratumoral therapy on mouse tumor weight and body weight at predose and postdose. Criteria for therapeutic activity: T/C (%), optimal growth inhibition <50 = effective. (E) Western blotting of CDK2 in each tumor derived from xenografts. (F) CD11b expression of cells derived from each tumor. (G-I) shCDK2 prolongs the survival of NOD/SCID mice inoculated with HL60 cells. (G) The population of human CD45⁺ and mouse CD45⁻ (hCD45⁺mCD45⁻) leukemia cells in the peripheral blood of NOD/SCID mice are presented. (H) The fluorescence-activated cell sorting–purified hCD45⁺ leukemic blasts in the mouse peripheral blood displayed by blood smear analysis on day 40 after transplantation. (I) The survival times of NOD/SCID mice were recorded. (J-K) shCDK2 prolongs the survival of NSG mice inoculated with PDX-AML blasts (n = 6). (J) The population of hCD45⁺mCD45⁻ leukemia cells in the peripheral blood of NSG mice were monitored weekly during the 3 months after transplant. (K) The survival times of NSG mice were recorded. (B,D-G) *P < .05, **P < .01, ***P < .001 vs scramble or as indicated. Data are shown as mean ± SD, n = 5 or 6. RTV, relative tumor volume; T/C, RTV_treatment/RTV_control × 100.