Therapeutic editing of hepatocyte genome in vivo

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Therapeutic editing of hepatocyte genome in vivo Marina Ruiz de Galarreta, Amaia Lujambio Journal of Hepatology Volume 67, Issue 4, Pages 818-828 (October 2017) DOI: 10.1016/j.jhep.2017.05.012 Copyright © 2017 European Association for the Study of the Liver Terms and Conditions

Fig. 1 Double strand breaks (DSBs) and DNA repair mechanisms. (A) DSBs (red scissors) can be repaired by non-homologous end-joining (NHEJ) repair mechanisms, giving rise to small insertions (violet) and deletions (light blue) that can disrupt gene expression. (B) Two DSBs can also be corrected by NHEJ and can generate inversions, inverting the DNA located between the two DSBs (in green, reverted), or deletions, by removing the DNA located between the two DSBs (in green). (C) DSBs can be repaired by homology directed repair (HDR) mechanisms in the presence of a donor template, allowing the incorporation of insertions (violet) or mutations (yellow star) that can disrupt gene expression. Black vertical bars indicate homology. Journal of Hepatology 2017 67, 818-828DOI: (10.1016/j.jhep.2017.05.012) Copyright © 2017 European Association for the Study of the Liver Terms and Conditions

Fig. 2 The four main gene editing platforms. The different nucleases (pale red) bind to DNA through protein-DNA interactions (meganucleases, ZFNs, TALENs) or protein-RNA-DNA interactions (CRISPR-Cas nucleases). ZFNs and TALENs contain protein domains that can bind 3 or 1 nucleotide(s), respectively, in a sequence specific manner. The nucleases induce different types of DSBs, represented by the red lines. DSB, double-strand break; nt, nucleotide; PAM, protospacer-adjacent motif; sgRNA, single guide RNA. The vertical black bars indicate homology. Journal of Hepatology 2017 67, 818-828DOI: (10.1016/j.jhep.2017.05.012) Copyright © 2017 European Association for the Study of the Liver Terms and Conditions

Fig. 3 Different liver diseases caused by hereditary mutations or viral infection can potentially be treated with gene editing approaches. Yellow stars represent non-synonymous mutations; the red “scissors” mark indicates the location of the DSB; the vertical black bars indicate homology; WT, wild-type; HDR, homology directed repair; NHEJ, non-homologous end-joining. Journal of Hepatology 2017 67, 818-828DOI: (10.1016/j.jhep.2017.05.012) Copyright © 2017 European Association for the Study of the Liver Terms and Conditions