Extended Data Fig. 10: AZD7648 improves gene targeting in B cells and HBECs.
a. Allelic distribution of mean WT, INDEL and HDR frequencies following gene editing at the CCR5 locus for knock-in of UBC-GFP-bGH-pA sequence in B cells with varying concentrations of AZD7648 using RNP/AAV6 gene editing (n = 2). HDR frequency was measured by ddPCR. WT and INDEL frequencies were measured through ICE analysis. Mean HDR to INDEL ratio is represented above the bars. b. Schematic for gene targeting at exon 1 of the CFTR gene for knock-in of SFFV-Citrine-pA sequence using RNP/AAV6 gene editing. c. Frequency of gene targeted cells as measured by flow cytometry for Citrine expression following CFTR-exon 1 gene editing (b) with or without AZD7648 treatment (0.5 μM) in HBECs (n = 4). d. Fold change in the frequency of CFTR gene targeted HBECs (c) with AZD7648 (0.5 μM) treatment relative to the untreated cells (n = 4). All data in c and d are shown as mean ± SEM.
