Extended Data Fig. 2: PABPC1 inhibition suppresses CML progression in BC stage and improves survival.
a, Schematic outline of conditional Pabpc1 knockout mouse model construction mediated by CRISPR/Cas9. b, Genomic iditification of mice with Pabpc1 deletion (n = 5). c, Representative FACS plots of GFP+ CD11b+ leukemia cells from PB of CML mice generated in Fig. 2a (n = 6 mice). d-e, Cell cycle distribution (d) and apoptosis rate (e) of LSK and LSC (LT-HSC) populations from primary recipients at day 21 after engraftment (in Fig. 2b) (n = 5 mice) f, Western blots showing protein levels in BMCs from Pabpc1 cKO mice (n = 3 mice). g, Apoptosis rate of BMCs in Pabpc1 cKO mice (n = 5 mice). h-i, Colony formation ability (h) and cell proliferation (i) of primary leukemia cells from (1#, 2#, 3#) CML-CP patients following PABPC1 inhibition (n = 3). j-k, Colony formation ability (j) and cell proliferation (k) of normal HSPCs from healthy donors following PABPC1 inhibition (n = 3). l, The generation of cell-derived xenograft (CDX) CML mouse model using K562 cells. m, Quantification of CD33+ CML cells in PB (left) and survival curves (right) of recipients (n = 6 mice). Data are shown as the mean ± SEM. P-values were determined by Student’s unpaired two-tailed t-test (d, e, g, h-k), one-way ANOVA with Bonferroni correction (m), and Kaplan–Meier survival analysis (m), ns P > 0.05, **P < 0.01, ***P < 0.001.