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. 2021 May 14;22:237–248. doi: 10.1016/j.omtm.2021.05.001

Figure 3.

Figure 3

Targeted human hematopoietic stem cells efficiently engraft in immunodeficient mice

(A) Percentage of GFP+ cells within the CD34+ compartment and within the HSC subset (CD34+CD38-CD90+), 48 h post-editing with RNP and GFP-AAV. n = 4; 4 healthy donor samples used. (B) Percentage of human cells in the bone marrow (BM) of immunodeficient NSG mice transplanted with AAV-GFP (white dots)- or AAV-coRPK (black dots)-edited cells, 1 and 3 mpt. Triangle dot corresponds to one animal transplanted with irradiated-CD34− cells only. Data are represented as mean ± SD (n = 7 mice in each group from 3 independent experiments). (C) Percentage of the hCD34+ cells within the human population (hCD45+) in mice analyzed in (B). Data are represented as mean ± SD (n = 7 mice in each group from 3 independent experiments). (D) Percentage of the hCD19+ cells within the human population in mice analyzed in (B). (E) Percentage of the hCD33+ cells within the human population in mice analyzed in (B). (F) Percentage of GFP+ cells within the human population (hCD45+) 1 and 3 mpt in mice analyzed in (B). (G) Percentage of GFP+ cells within the human progenitor cells (hCD34+) 1 and 3 mpt in mice analyzed in (B). Data are represented as mean ± SD (n = 7 mice in each group from 3 independent experiments). Significance was analyzed by non-parametric two-tailed Mann-Whitney test. ns, not significant.