In vivo and ex vivo assessment of engraftment and lineage proliferation over time and at necropsy
(A) Example of flow cytometric gating strategy utilized to determine total human engraftment and delineate human lineages in the PB and BM of humanized mice. (B) Total human engraftment and lineage-specific proportions over time in the PB of transplanted mice (mean ± SD). (C) Total human engraftment in BM and PB of transplanted mice at time of necropsy (mean ± SD). (D) Human lineage analysis in BM and PB of transplanted mice at time of necropsy (mean ± SD). (E) Example of flow cytometric gating strategy for determination of human hematopoietic stem and progenitor cell proportions within BM of transplanted mice at time of necropsy, including assessment of the hematopoietic stem cell-enriched CD90+CD45RA– subpopulation. (F) Human hematopoietic stem and progenitor cells, and hematopoietic stem cell-enriched CD90+CD45RA– cells within the BM of transplanted mice at time of necropsy (mean ± SD). (G) CFUs grown from mouse BM cells (mean ± SD). BCL11A, BCL11A-ee; HBG, HBG-113; Seq, sequential; Sim, simultaneous.