Fig. 3
Specificity of RNA editing mediated by AAV-delivered circ-arRNA in NHPs. A AEI rate for the control and 3 × 1013 vg/kg dosage groups. Each group includes two biological replicates, and each replicate consists of 3 technical replicates; mean ± SD. B FPKM (Fragments Per Kilobase of exon model per Million mapped fragments) values for ADAR1, ADAR2, and ADAR3 in the Ctrl and 3 × 1013 vg/kg dosage groups. Each group contains two biological replicates, and each replicate includes 3 technical replicates; mean ± SD. FPKM values were calculated using the STRINGTIE tool. C The relative expression levels of PPIA transcripts measured by qPCR in the Ctrl and 3 × 1013 vg/kg dosage groups. D Differential gene expression analysis comparing the Ctrl group and 3 × 1013 vg/kg dosage group, based on RNA-seq data at the transcriptome level. FPKM values were calculated using the STRINGTIE tool. Pearson’s correlation coefficient analysis was used to assess the global differential gene expression. The on-targeting site (PPIA) is highlighted in red. E Transcriptome-wide analysis of potential off-target editing between the Ctrl and 3 × 1013 vg/kg dosage groups. The pink dots represent off-target sites located in the UTR region. The blue dots represent off-target sites causing synonymous mutations, and the green dot represents an off-target site causing missense mutation. F Editing rates of the target site (0) and three severe bystander off-target sites (− 28, − 23, and 5). Each point represents data from a single liver lobe; mean ± SD. Student’s T test was used for all statistical comparisons between the treatment and control groups