Tissue engraftment and reconstitution of the BM stem cell compartment in mice transplanted with NP- versus EP-edited human CD34+ cells
(A) Frequency of human chimerism in the BM, spleen, and thymus. (B) Frequency of engrafted human CD34+ cells (left y axis) and the HSC-enriched CD34+CD90+ subset (right y axis) engrafted in the BM. (C and D) Total CFC potential (left graphs) and quantification of erythroid, myeloid, and erythromyeloid colonies (right graphs) of human (C) CD34+ and (D) CD34+CD90+ cells. CFU, colony-forming unit; CFU-M, CFU monocyte/macrophage; CFU-G, CFU granulocyte; CFU-GM, CFU granulocyte/monocyte/macrophage; BFU-E, burst-forming unit erythrocyte; CFU-Mix, CFU containing a mix of erythroid and myeloid cells. (E) Quantification of the PCR-based assessment of CD33 exon 2 knockout (CD33ΔE2) in PB and BM at week 20 shown in Figure S4D. (F) Genomic analysis of the CD33 genotype (+/+, wild type; +/−, heterozygous knockout; −/−, homozygous knockout) of individual CD34+- and CD34+CD90+-derived colonies (representative gel pictures shown in Figures S4E and S4F). Statistics: (A and B) mean ± SEM, one-way ANOVA; (C and D) mean ± SD, one-way ANOVA.