Skip to main content
. 2020 Jul 3;11:3327. doi: 10.1038/s41467-020-17148-x

Fig. 6. Improved macrophage differentiation of GCase-targeted HSPCs in NSG-SGM3 mice.

Fig. 6

a Human cell engraftment 16-weeks post-transplantation in the bone marrow (BM), spleen (SP), and peripheral blood (PB) in transplants using CD68S-GCase-P2A-Citrine-targeted cells (n = 5 mice). b Modified allele frequency from engrafted CD68S-GCase-P2A-Citrine-targeted cells in the same tissues (n = 5 mice). c Percent human B-cell (CD19+), myeloid (CD33+), and monocyte (CD14+) populations in BM, SP, and PB shown in white. Citrine-positive cells in each population are shown in green (n = 5 mice). d Representative FACS plots showing gating strategy for mouse and human CD45+, CD45+/CD11b+, and CD45+/CD11b+/Citrine populations in macrophage preparations from lung, peritoneum, and liver. e Percent human CD45+ and human CD45+/Citrine cells in the same preparations (n = 5 mice). f Percent human CD45+/CD11b+ and human CD45+/CD11b+/Citrine cells in the same preparations (n = 5 mice). g Fold GCase activity in human Citrine+ cells compared to human Citrine cells in BM, SP, and lung (n = 3 mice). h Modified allele frequency in human Citrine+ cells (green) compared to human Citrine cells (white) in BM, SP, and lung in the same cells. a, b Median shown. c, eg Data shown as mean ± SD. Source data are provided as a Source Data file.