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. 2022 Feb 28;30(6):2186–2198. doi: 10.1016/j.ymthe.2022.02.026

Figure 4.

Figure 4

Long-term peripheral blood chimerism in mice transplanted with NP- versus EP-edited human CD34+ cells

(A) Cell viability and (B) reduction of CD33 cell-surface expression determined 72 h after NP- and EP-mediated editing of human CD34+ cells. Untreated cells were used as a control. (C and D) Longitudinal flow cytometric assessment of (C) human chimerism and (D) CD14+ monocytes in the PB in mice transplanted with untreated, EP-RNP-, and NP-RNP-edited human CD34+ cells. (E) Human multilineage engraftment in the PB at week 20 post-transplant. (F) PCR validation of CD33 editing in human cells in the PB. (G) Longitudinal monitoring of human CD14+ monocytes in the PB lacking CD33 exon 2 on the cell surface (CD33ΔE2) via flow cytometry. (H) Quantification of PCR bands in (F). Statistics: (A and B) mean ± SD, one-way ANOVA.