Figure 3.

Editing of clinically relevant genomic loci by RNP delivery of high-fidelity SpCas9 variants in CD34+ cells

Primary CD34+ cells were electroporated with WT SpCas9, HiFi Cas9, or K562D RNPs targeting the CCR5, HBG, and HBB loci. Targeted deep sequencing analyses were performed 6 days post-transfection at the on-target sites (A) or at reported identified off targets (B). The off-/on-target ratios for WT SpCas9 and the two SpCas9 high-fidelity variants (HiFi Cas9 and rCas9HF) calculated from (A) and (B) are reported in Figure S2B. Statistical significance was assessed using paired t test; data reported as mean ± SEM for n = 3 biologically independent replicates.