Fig. 7: Administration of miRisten effectively inhibits AML propagation and leukemia-initiating activity in inv(16) AML PDX model.

a Schematic of experimental design. Inv(16) AML PDX was established by directly injecting T cell-depleted primary AML cells (1 × 10⁶ cells/mouse) i.f. into irradiated NSGS and subsequently expanded into larger cohorts by secondary transplantation via i.v. injection. After 8 weeks, PDX mice were treated with SCR control (20 mg/kg/dose, i.v., daily) or miRisten (20 mg/kg/dose, i.v., daily) for 3 weeks and followed by assessment of human cell engraftment in BM and spleen. b Representative FACS plots showing gating strategy and frequency of hCD45⁺ cells in BM of mice treated with SCR control (top) or miRisten (bottom) for 3 weeks. c Representative FACS plots showing gating and frequency of hCD34⁺/CD45⁺ cells in BM of mice treated with SCR (top) or miRisten (bottom) for 3 weeks. d Frequency of hCD45⁺ AML cells in SCR (black) vs. miRisten (red) treated mice (n = 9/group) BM (p = 0.0222) or spleen (SP). e Frequency of hCD34⁺/CD45⁺ AML cells in SCR (black) vs. miRisten (red) treated mice (n = 9/group) BM (p = 0.01) or SP (p = 0.0385). f The Kaplan–Meier survival curve of mice treated with SCR (black line; n = 6; median survival 117 days) or miRisten (red line; n = 6; median survival 136 days; p = 0.0161) for 3 weeks starting at 8 weeks after transplant. Dotted line with gray shade indicates treatment window. g The Kaplan–Meier survival curve of second transplant recipients treated with SCR (black line; n = 5; median survival 137 days) or miRisten (red line; n = 5; median survival 199 days; p = 0.0127). The statistical significance for f, g was determined using log-rank (Mantel–Cox) test. Each dot in d, e represents result from an individual mouse and data are presented as the mean ± SEM; statistical significance was determined using two-tailed Student’s T-tests (*p < 0.05; **p < 0.01; ***p < 0.001).