Figure 4.
HBA2-deleted and βAS3KI HSPCs engraft NSG mice and maintain their multilineage potential. (A) Schematic representation of engraftment experiments. (B) Percentage of human CD45+/HLA-ABC+ cells in hematopoietic organs of mice. BM, bone marrow; PB, peripheral blood; SP, spleen. Black lines indicate mean. (C) InDel efficiency in PB of RNP-engrafted mice at different timepoints (mean ± SD; n = 2-4). (D) HBA2 copies in RNP-treated HSPCs at day 0 (injection) and in BM of engrafted mice at week 16. Black lines indicate mean; red line indicates the number of expected HBA2 alleles in untreated HSPCs. (E) GFP+ cells in PB of transplanted mice over time. GFP is expressed as percentage of CD45+ cells; line indicates mean.

HBA2-deleted and βAS3KI HSPCs engraft NSG mice and maintain their multilineage potential. (A) Schematic representation of engraftment experiments. (B) Percentage of human CD45+/HLA-ABC+ cells in hematopoietic organs of mice. BM, bone marrow; PB, peripheral blood; SP, spleen. Black lines indicate mean. (C) InDel efficiency in PB of RNP-engrafted mice at different timepoints (mean ± SD; n = 2-4). (D) HBA2 copies in RNP-treated HSPCs at day 0 (injection) and in BM of engrafted mice at week 16. Black lines indicate mean; red line indicates the number of expected HBA2 alleles in untreated HSPCs. (E) GFP+ cells in PB of transplanted mice over time. GFP is expressed as percentage of CD45+ cells; line indicates mean.

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