Fig. 3: Secondary transplantation reveals a reduction of leukemic stem frequency upon DNMT3A^R882H correction.

A: Schematic of secondary transplantation experiments. Human leukemic cells are harvested from primary recipients 4 months after gene-editing and then transplanted in limiting dilution into secondary host animals. B: Quantification of secondary limiting dilution assay results for patient specimen SU372.C. Estimated LSC frequencies and test statistics are indicated. C: Quantification of secondary limiting dilution assay results for patient specimen SU540. None of the mice receiving corrected (DNMT3A^WT/WT) leukemic cells showed engraftment at the doses indicated. Estimated LSC frequencies and test statistics are shown. D: VAF in human genomic DNA isolated from secondary recipients for recurrent somatic mutations found in patient samples utilized for secondary transplantation studies. E: FLT3-ITD PCR from human genomic DNA isolated from secondary recipients.