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. 2023 Nov 30;52(2):977–997. doi: 10.1093/nar/gkad1125

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© The Author(s) 2023. Published by Oxford University Press on behalf of Nucleic Acids Research.

This is an Open Access article distributed under the terms of the Creative Commons Attribution-NonCommercial License (https://creativecommons.org/licenses/by-nc/4.0/), which permits non-commercial re-use, distribution, and reproduction in any medium, provided the original work is properly cited. For commercial re-use, please contact journals.permissions@oup.com

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Figure 6. — Improving in vivo editing efficiency through optimization of the dosing regimen. (A) 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).