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[Preprint]. 2024 Oct 25:2024.10.23.619839. [Version 1] doi: 10.1101/2024.10.23.619839

Figure 1. Different DNA editing outcomes generated by the three ABE variants.

Figure 1.

(A) DNA context around the nonsense mutation of rd12 mice. The arrow indicates the sgRNA for NG-ABEs and colors indicate target adenosine (red), bystander adenosine (blue), bystander cytosine (yellow), and PAM (green). Nucleotide number indicates position, counting PAM as position 21–23. (B) Schematic drawing of the dual-AAV vectors for ABE delivery. CMV and U6 are promoters. Npu-C and Npu-N indicate C- and N-intein from N. punctiforme, respectively. (C) Schematic showing outline of in vivo experiments. (D) High-throughput sequencing results of the nonsense mutation region in the genomic DNA isolated from RPE tissue of no injection (No IJ) (n = 11), ABE8e-treated (n = 6), ABEmax-treated (n = 8), and ABE8eWQ-treated mice (n = 7). The split-AAV strategy was utilized to deliver ABEs, and each component of split ABEs was packaged into AAV2/9. Error bars indicate mean ± s.e.m.