Extended Data Figure 2. BE1 is capable of correcting disease-relevant mutations in vitro.

a, Protospacer and PAM sequences (red) of seven disease-relevant mutations. The disease-associated target C in each case is indicated with a subscripted number reflecting its position within the protospacer. For all mutations except both APOE4 SNPs, the target C resides in the template (non-coding) strand. b, Deaminase assay showing each dsDNA 80-mer oligonucleotide before (–) and after (+) incubation with BE1, DNA isolation, and incubation with USER enzymes to cleave DNA at positions containing U. Positive control lanes from incubation of synthetic oligonucleotides containing U at various positions within the protospacer with USER enzymes are shown with the corresponding number indicating the position of the U. Editing efficiencies were quantitated by dividing the intensity of the cleaved product band by that of the entire lane for each sample. For gel source data, see Supplementary Figure 1.