Fig. 2
αCD33-mAB-P/P-siRNA-mediated RNAi inhibits target gene expression and decreased growth of DNMT3A-mutant AML cells in vitro and in vivo. A Western blot analysis of treated OCI-AML2 cells. B–D Colony formation assay of DNMT3A mutant cell lines OCI-AML2 (B, n = 6) OCI-AML3 (C, n = 6) and DNMT3A-wild type KG1 cells (D, n = 3). There is a significant decrease in colony growth due to αCD33-mAB-P/P-DNMT3A-siRNA treatment in OCI-AML2 and OCI-AML3 (B-C, in contrast to control-siRNA), but not in KG1 (D). Significance: *, p < 0.05, 2-tailed T-test. Means plus SD of three independent experiments. E. Schematic overview about in vivo treatments after subcutaneous (s.c.) injection of 1 × 107 AML cells in CD1-nude mice. Mice were treated intraperitoneally (i.p.) with PBS, αCD33-mAB-P/P-control (cntr)-siRNA or αCD33-mAB-P/P-DNMT3A-siRNA three times weekly. F,G Tumor growth curves of OCI-AML2 (F) and KG1 (G). Growth of OCI-AML2 tumors was significantly delayed due to systemic αCD33-mAB-P/P-DNMT3A-siRNA treatment in contrast to control-siRNA treatment and PBS treatment (G), whereas KG1 tumors (DNMT3A wild type) did not show significant differences in tumor growth (G). H,I Tumor weight of isolated OCI-AML2 (H) and KG1 (I) tumors. Shown are means plus SD: T-test *, p < 0.05. α, anti