Fig. 1

Optimization of arRNA for use in non-human primates. A NGS results showing the editing efficiency at target sites within PPIA transcripts in FRhK-4 cells derived from monkey; n = 2, mean ± SD. B NGS results showing the editing efficiency at both the target and bystander adenosine sites within PPIA transcripts in monkey FRhK-4 cells. The target adenosine is highlighted by a black arrow, while three severely edited bystander adenosines are denoted by black triangles; n = 2, mean ± SD. C NGS results exhibiting the editing efficiency at the target and three severely edited bystander adenosine sites of PPIA transcripts in monkey FRhK-4 cells. ΔA denotes circ-arRNA with corresponding uracil deletion at bystander adenosine sites; n = 2, mean ± SD. D NGS results showing the fold change in targeted editing rates when arRNA is driven by CAG (Pol II), U6 (Pol III), or a tandem combination of U6 (Pol III) and CAG (Pol II). Editing rates are normalized to the U6 promoter; n = 2, mean ± SD. E Editing efficiency of the target adenosine in primary hepatocytes from monkeys post-AAV8 infection at days 4, 7, 14, and 21 post-infection; n = 3, mean ± SD. F Editing of bystander off-target sites at days 4 and 7 post-AAV8 infection in monkey primary hepatocytes. The target adenosine is indicated by a black arrow, while three severely edited bystander adenosines are denoted by black triangles; n = 3, mean ± SD