Fig. 6: dBP2b and dAP1b8e base editors can induce high base editing efficiencies without indel mutations in primary myoblasts.
From: Precise base editing without unintended indels in human cells and mouse primary myoblasts
a Construction of dBP2b and dAP1b8e. b Illustration of premature stop codon introduction by C-to-T conversion in exon 20 of the Dmd gene or rescue by A-to-G transition in exon 20 of Dmd. c Nucleotide substitution frequencies of CBE variants (BE3, AncBE4max, AncdBE4max, and dBP2b) in C2C12. d Indel frequencies of the BE3, AncBE4max, AncdBE4max, and dBP2b variants. e A-to-G conversion frequencies of ABE variants (ABE8e, dABE8e, and dAP1b8e) in Dmd+/Q871* NIH3T3 cells. f Indels were not generated by any ABE variants. g Schematic outline of the mouse primary myoblast experiment. Primary myoblast cells were obtained from WT or Dmd Q871* neonatal skeletal muscle. The base editor and sgRNA-containing vectors were delivered to the cells on the second day of differentiation. h–j Identification of base substitutions and indel frequencies on days 3, 5, and 10 after transfection of CBE variants (AncBE4max, AncdBE4max, and dBP2b) and ABE variants (ABE82, dABE8e, and dAP1b82) in myoblast cells. k All ABE variants showed no indels. Control indicates the untreated group. The data are shown as the mean ± S.D. of three independent experiments. Each dot represents an individual target experiment.
