Skip to main content
[Preprint]. 2023 Mar 23:2023.03.20.533459. [Version 2] doi: 10.1101/2023.03.20.533459

Figure 6. Improving in vivo editing efficiency through optimization of the dosing regimen. a.

Figure 6.

). Schematic representation of the experimental process to test a repeat crRNA dosing regimen. RO, retro-orbital injection. b). Comparing the editing efficiency of AAV-SpyCas9-ABE-tracrRNA co-delivered with either a single 80 mg/kg dose, or three consecutive daily injections 26 mg/kg dose of C20+PO07-GalNac targeting the mouse Pcsk9 gene. The editing efficiency was measured by targeted amplicon deep sequencing of mouse liver genomic DNA (n = 3 mice per group). The single-dose data are the same as those shown in Figure 5b. Data represent mean ± SD; ****, P < 0.0001 (two-way ANOVA).