A humanized Animal Model Predicts Clonal Evolution and Therapeutic Vulnerabilities in Myeloproliferative Neoplasms

We performed single cell RNA sequencing to ensure that the engrafted MF cells in NSGS mice retained the molecular properties of the patients cells. ScRNAseq profiles from peripheral blood mononuclear cells (PBMCs) from two independent cord blood and MF patient samples were compared to the engrafted hCD45+ cells from the bone marrow of NSGS mice at 12-weeks post-transplant. Overall design: Human CD45+ cells were purified by FACS from CB- or MF-derived mononuclear cells as well as from NSGS xenografs. Purified cells were resuspended at 1,200 cells per uL in PBS + 0.04% BSA. cDNA synthesis, QC, and library sequencing are described in the manuscript in detail.