Figure 1.
Gene editing of human CD34+ cells after RNP electroporation. (A) Schematic depiction of the genome editing targets in chromosomes 2 and 11. (B) Sequence of the optimal sgRNAs used for the downstream assays. Overlined is the binding sequence of the transcription factors GATA1, LRF, and BCL11A, respectively. (C) Percentage of introduced indels 2 days post-RNP electroporation. (D) Frequency of microhomology-mediated end joining (MMEJ) within the different indels. (E) Most abundant genotypes (representing >2% of the alleles) post–genome editing for the 3 loci. All plots represent data from at least 4 different CD34+ cell donors. Values are represented as means ± standard error of the mean (SEM). ***P ≤ .0001, *P ≤ .05 vs untreated (untr); ††P ≤ .001, †P ≤ .05 vs HBG-197, ‡P ≤ .05 vs HBG-115 (unpaired Student t test).