Extended Data Fig. 6: Human CD34+ HSPC-derived VEXAS model recapitulates features of human disease.
From: Independent mechanisms of inflammation and myeloid bias in VEXAS syndrome
a, Schema for engineering UBA1M41T/y HSPCs and characterizing their effect on hematopoiesis via transplantation into NBSGW recipient mice. Created in BioRender. Gitlin, A. (2025) https://BioRender.com/2vd7lid. b, Wright-Giemsa stain of human HSPCs 6 days after electroporation and quantification of vacuolated cells (red arrows) per high power field (HPF) (n = 6-7 HPF per genotype, counted manually). c, Immunoblots of human HSPCs of indicated genotypes 6 days after electroporation. d, Crispresso2-based27 quantification of % M41T alleles detected by amplicon sequencing of a region surrounding Met41 in UBA1 from genomic DNA of human CD45+ cells sorted from bone marrow of NBSGW mice 4 weeks after transplantation with HSPCs of the indicated genotypes (n = 3 mice per genotype). Each dot represents genotyping quantification from an individual mouse. Bars in b and d represent mean; error bars represent SEM. P value in b was calculated using unpaired, two-sided t-test. Results in b and c are representative of 2 independent experiments.
