Extended Data Fig. 2: Prime editor 2 corrects the disease mutation and phenotype in Fah^mut/mut mice.

a, Maps of vectors encoding PE2 components and a schematic representation of the experiments. Fah^mut/mut mice underwent injection of plasmids encoding prime editor 2 components (that is, prime editor 2 and pegRNA) and were kept on water containing NTBC for 7 days. The day on which NTBC was initially withdrawn is defined as day 0. The mice were again provided with NTBC for five days, from day 7 to day 12, after the initial withdrawal of NTBC at day 0. At 60 days, the PE2-treated mice were euthanized and analyzed. Abbreviations in the vector maps are defined in the Supplementary Figure 2 legend. b, Body weight of Fah^mut/mut mice injected with PE2 or phosphate-buffered saline (Saline, control). Body weights were normalized to the pre-injection weight. The number of mice n = 5 for the PE2 group and n = 3 for the saline group. Data are mean ± s.e.m. c, The level of wild-type Fah mRNA in the liver measured by quantitative RT-PCR using primers that hybridize to exons 8 and 9. WT, wild-type mice; Mut, Fah^mut/mut mice; PE2, Fah^mut/mut mice injected with plasmids encoding PE2 components. The number of mice n = 3 (WT), 3 (Mut), and 5 (PE2). **P = 0.0032. d, H&E staining (upper panels) and immunofluorescent staining for FAH protein (lower panels) in liver sections. Scale bars, upper panels, 100 μm; lower panels, 200 μm.