Gene expression analysis of human sickle cell disease (SCD) and healthy donor (HD) CD34+ hematopoietic stem/progenitor cells (HSPCs) treated with CRISPR/Cas9 targeting the HBG promoters or untreated samples
We have investigated gene expression changes occurring in samples electroporated with Cas9 ribonucleoprotein (RNP) complexes targeting the -196 or the -197 nucleotide upstream of the HBG promoters or the unrelated AAVS1 locus Overall design: We performed RNA-seq analysis in SCD and HD CD34+ HSPCs (3 donors) that were preactivated for 2 days, then electroporated with Cas9 RNP containing guide RNA targeting the -196 or the -197 nucleotide upstream of the HBG promoters or the unrelated AAVS1 locus, and then cultured for 2 additional days. Control untreated cells were also cultured for a total of 4 days.