Fig. 5: Gene editing applications of gTBE and gCBE.

a Principle for exon skipping with base editors. b Bar plots showing the numbers of sgRNA candidates targeting the splicing sites in 16 genes by different base editors. gCBE, gCBEv2; gGBE, gGBEv6.3; gTBE, gTBEv3. The 16 genes are AGT, ANGPTL3, APOC3, B2M, CD33, DMD, DNMT3A, HPD, KLKB1, PCSK9, PDCD1, PRDM1, TGFBR2, TRAC, TTR, and VEGFA. c Venn diagram showing the distribution of sgRNAs for 4 base editors in (b). d Schematic diagram illustrating sgRNA candidates specifically targeting SD or SA sites in human DMD with gTBEv3 (red lines) or gCBEv2 (black lines), but not ABE or CBE. e Schematic diagram illustrating the skipping of human DMD exon 45 induced by gTBE-induced disruption of the splicing donor site. f On-target base editing efficiency for gTBEv3 targeting the splicing donor site of humanized DMD exon 45 in mouse embryos (mean ± s.e.m., n = 20). g DNA sequencing chromatograms from wild-type (WT) and representative embryos co-injected with gTBEv3 mRNA and sgRNA targeting the SD site of human DMD exon 45. Source data are provided as a Source Data file.