Skip to main content
. 2024 Jul 15;15:5927. doi: 10.1038/s41467-024-50340-x

Fig. 2. ABE system efficiency mediated splice site mutation in vitro.

Fig. 2

a The sgRNA screening scheme of adenine base editor (ABE) mediated editing of splicing site in HEK293T cells. b A-to-G conversion rate for 10 sgRNAs targeting exon 50 of DMD gene. c Comparison of the editing efficiencies for ABE systems split with various intein sequences. d Schematic of the AAV vectors for ABE1 and ABE2 variants. TadA8e, Cas9n split by Rhodothermus marinus (Rma) intein and the sgRNA6 were packaged into 2 separated AAV particles. A muscle-specific promoter Spc5-12 was used to drive TadA8e-Cas9n-N or Cas9n-C. ABE1 only contained two copies of sgRNA in Cas9n-C, but ABE2 contained two and one copies in Cas9n-C and TadA8e-Cas9n-N, respectively. Each dot represents individual biological replicates. Data are presented as mean ± s.d (n = 3 independent biological replicates). Significance is indicated by asterisk and determined using unpaired two-tailed Student’s t test. Source data are provided as a Source Data file.