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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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Gene targeting of adult FIX KO mice with gene-targeting strategy coupled to SaCas9. (A) Experimental plan. FIX KO adult mice were injected at 2 months of age (P60) with 1.0E11 vg/mouse of AAV8-SaCas9 and 5.0E11 vg/mouse of AAV8-donor-hFIX. Blood was collected 1 and 2 months after treatment. (B) Plasma hFIX levels in mouse transduced with SaCas9 and donor-hFIX at post-natal (P) day 2 (n = 15) and 60 (n = 8). hFIX concentration obtained from blood samples at months 1, 2, and 4 (P2 treatment) and 2, 3 and 4 months (P60 treatment). 5,000 ng/ml corresponds to the normal FIX plasma levels in healthy individuals in the human population. (C) Tail-bleeding assay. Bleeding time was evaluated in treated adult FIX KO mice (n = 8) and compared to untreated WT (n = 10) and FIX KO (n = 8) mice. Data are shown as mean ± SEM and statistically analyzed by one-way ANOVA with Tukey’s multiple comparison test.