Fig. 5. TJ-PE rewrites a correction exon in mouse liver.

a Diagram of Fah splicing before and after correction by TJ-PE. b A diagram of the TJ-PE strategy at Fah locus. c TJ-PE treatment rescues body weight after NTBC withdrawal. The body weight ratio is normalized to day 0 of NTBC withdrawal. NC: treated with Saline. d Schematic of the split-intein dual AAV8 system and tail vein injection experimental timeline. Four-week-old tyrosinemia I mice were injected with a total of 2 × 10¹² vg AAV8. e Representative FAH IHC images. Scale bars, 100 μm. Mice treated with saline were used as negative controls. The lower panel of AAV is a high-magnification view (box with black line). f Quantification of FAH⁺ hepatocytes by IHC six weeks after AAV injection. Data represent mean ± SD. (n = 4 mice for each group).