Fig. 6: Applications including base editing and modulation of the mini variants in vivo.

a Schematic representation of Vx and mini-Vx. Vx: REPAIRx, mini-Vx: mini-REPAIRx. b A-to-I editing efficiency of Vx and mini-Vx with endogenous transcripts in HEK293T cells, as analyzed by deep sequencing. The data are presented as the mean ± SD. Two-way analysis of variance (ANOVA) followed by Sidak’s multiple comparisons. ns not significant. (n = 3 biological replicates). Source data are provided as a Source data file. c Quantification of the percentage COG3 edited at the targeted adenosine (blue triangle) and neighboring sites by Vx and mini-Vx. Source data are provided as a Source data file. d Quantification of the percent of GLI1 editing at the targeted adenosine (blue triangle) and neighboring sites by Vx and mini-Vx. Source data are provided as a Source data file. e Experimental scheme. f Quantification of Pcsk9 mRNA levels in the livers from AAV8-injected mice (n = 6) and noninjected mice (n = 6). The data are presented as the mean ± SD. P values are by Wilcoxon matched-pairs signed-rank test. *P < 0.05. Source data are provided as a Source data file. g Quantification of Pcsk9 protein levels in the livers from AAV8-injected mice. h Quantification of total serum cholesterol levels at 3 weeks (noninjected, n = 6; crPcsk9, n = 6). i Serum ALT was quantified in noninjected mice (n = 6) and AAV8-crPcsk9-injected mice (n = 6). j Serum AST was quantified in noninjected mice (n = 6) and AAV8-crPcsk9-injected mice (n = 6). h–j The data are presented as the mean ± SD. P values are by Wilcoxon matched-pairs signed-rank test. *P < 0.05, ns not significant. Source data are provided as a Source data file.