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

Fig. 4. GCase-targeted HSPCs sustain long-term hematopoiesis.

Fig. 4

a Total number of colonies formed from mock, Citrine+ and Citrine– SFFV and CD68S-driven constructs (n = 4 biologically independent human donor samples for Mock and SFFV and n = 2 for CD68S). b Distribution of phenotypes of colonies formed. Erythroid progenitors (burst-forming unit-erythroid or BFU-E (red)) and colony-forming unit-erythroid or CFU-E (blue), granulocyte-macrophage progenitors (CFU-GM, green), and multi-potential granulocyte, erythroid, macrophage, megakaryocyte progenitor cells (CFU-GEMM, purple) (n = 4 biologically independent human donor samples for Mock and SFFV and n = 2 for CD68S). c Primary human engraftment (16 weeks) in the bone marrow in transplants using CD68S-GCase-targeted and CD68S-GCase-P2A-Citrine-targeted cells (blue circles: 0.25E6, green: 1E6, and red: 2E6 cells transplanted; n = 31, 33 mice). d Primary human engraftment in the spleen. e Targeted allele frequency in CD68S-GCase- and CD68S-GCase-P2A-Citrine-targeted cells before transplantation (Pre-Tx) and 16-weeks post-transplantation (Post-Tx) in engrafted human cells in the bone marrow of mice with human chimerism >1% (n = 29, 31 mice). f Secondary human engraftment (32 weeks) in the bone marrow (n = 8, black: CD68S-GCase-P2A-Citrine, white: CD68S-GCase). Note, three mice have chimerism <1%. g Targeted allele frequency before (Pre-Tx) and after transplant (Post-Tx) in the bone marrow cells of secondary mice. a–b Data shown as mean ± SD. c–g Median shown. Source data are provided as a Source Data file.