Fig. 1.

CRISPR/Cas9-mediated isoform expression of GATA1 at single cell level. a Experimental workflow for single cell in vitro differentiation assay and near-clonal xenotransplantation into mice. b Two gRNAs targeting the 5′ and 3′ end of exon 2 of GATA1 led to the NHEJ-mediated dropout of this exon, resulting in the exclusive expression of GATA1-Short. c HDR-mediated mutation of the alternative start site from ATG to CTC using a single gRNA and a single-strand DNA template, resulting in the exclusive expression of GATA1-Long