Fig. 2 | Nature Communications

Fig. 2

From: Gene correction for SCID-X1 in long-term hematopoietic stem cells

Fig. 2

Normal hematopoietic reconstitution from IL2RG cDNA targeted CD34HSPCs. a Timeline of primary (1°) and secondary (2°) human transplants into sub-lethally irradiated NSG mice. CD34HSPCs are derived from umbilical cord blood of healthy male donors. Adult mice transplanted intra-femoral (IF) with either WT CD34+HSPCs (white circles) or mock targeted (yellow circles) or RNP only (black circles) or un-selected IL2RG cDNA targeted (blue-black circles) HSPCs. Three – 4 days old NSG pups transplanted intra-hepatic (IH) with either mock or IL2RG targeted HSPCs. b Combined IF and IH human cells engraftment (hCD45+HLA A-B-C+) 16 weeks after 1° human transplant into indicated organs. c %IL2RG cDNA targeted HSPCs within human graft in indicated organs,  quantified by ddPCR. BM (n = 24 mice), SP (n = 24 mice), PB (n = 6 mice) (***p = 0.0008, one-way ANOVA). d Percent human engraftment in indicated organs as in (b) 16 weeks post 2° human CD34HSPCs transplant into adult NSG mice. *p-value SP-IH = 0.025, *p-value BM-IF = 0.043 (Welch’s t-test). e %IL2RG targeted HSPCs quantied by ddPCR 32 weeks after engraftment. Median shown. BM bone marrow, SP spleen, PB peripheral blood. Source data are available in the Source Data file

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