Fig. 4
Xenograft assay. a Schematic of the workflow of mouse xenograft experiment. b Flow cytometry analysis of cells following CRISPR transfection demonstrating transfected (positive for GFP) CD34+ HSPCs. Gating strategy is shown in Supplementary Fig. 10b. c Flow cytometry plots of harvested bone marrow from xenograft mice gated for live cells demonstrating hCD45 expression. Gating strategy is shown in Supplementary Fig. 10c. d Gel electrophoresis image of genomic DNA extracted from human cells obtained from xenograft mouse analyzed by PCR demonstrating genome-edited bands. e Characterization of deletion break points by sequencing (co-ordinates from Hum Mar 2006 (NCBI36/hg18) Assembly) f Morphological analysis of hematopoietic colonies grown in methyl cellulose which were generated by HSCs harvested from secondary transplant mice (two independent mice)
