Figure 1.

Screening of guide sequences. (A) Guide sequences with editor type and targeting site information. Red, targeted bases in critical motifs; green background, optimal targetable window (also indicated by the numbers above the column). A CRISPR/Cas9 plasmid targeting the TGACCA motif in the HBG1/2 promoter was used as a positive control. A CBE plasmid targeting the CCR5 coding region was included as a negative control (sgNeg). (B) HUDEP-2 cells were transfected with plasmids expressing guide sequences and the BEs listed in panel A. For example, p.sgBCL-1 indicates the plasmid-expressing guide sequence sgBCL-1. γ-Globin expression was measured by flow cytometry 4 days after transfection (4dpt) and 6 days after in vitro erythroid differentiation (Diff 6d). Nontr, nontransduced. (C) The percentage of base conversion detected at 4dpt by Sanger sequencing. Data are the mean ± SD of 3 biological replicates.