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. 2022 Mar 11;4:785698. doi: 10.3389/fgeed.2022.785698

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Copyright © 2022 Lisjak, De Caneva, Marais, Barbon, Biferi, Porro, Barzel, Zentilin, Kay, Mingozzi and Muro.

This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.

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Evaluating the genome targeting rate, the presence of viral genome copies and hFIX protein in hepatocytes. (A) Genome targeting rate was evaluated in neonatal and adult-treated mice by ddPCR of liver genomic DNA (from Figures 2, 5); (B,C) rAAV8-SaCas9 and rAAV8-donor-hFIX viral genome copies analysis from FIX KO mice treated at P2 and P60 (from Figures 2, 5); (D) Western blot analysis of hFIX protein in liver extracts. Mice treated at P2 and P60, together with untreated WT and KO were analyzed. Human plasma was used as a positive control; (E) Western blot was quantified and normalized with housekeeping GAPDH protein.