Figure 1.
![Figure 1. In vivo gene targeting of the FIX locus in FIX-KO mouse liver by AAV.SaCas9. Schematic diagrams of the mouse FIX locus show the SaCas9.sgRNA3 target site located in the 5′ end of exon 2; the AAV donor vector that contains U6-sgRNA3 and partial cDNA of codon-optimized, Padua-containing hFIX cDNA (hFIXco-Padua [exon 2-8]) followed by a polyA and flanked by homology arms; the modified mFIX locus after homologous recombination; the chimeric FIX mRNA transcribed from the gene-targeted FIX locus; and the translated chimeric FIX protein. Blue boxes indicate mFIX exons or exon-derived mRNA or protein sequences, and orange boxes indicate codon-optimized hFIX cDNA (exon 2-8), mRNA, or protein.](https://ash.silverchair-cdn.com/ash/content_public/journal/blood/133/26/10.1182_blood.2019000790/3/bloodbld2019000790f1.png?Expires=1735576619&Signature=Nkb~JCrM1N93hLyLmYxz~rg~-bhH35hkfPtmeXsvVseOqdp-gLZbmmimNyntW1NJo4TtHX~W2bVvXufTU4O5p05G4X6zMVPY3s2u~zf-rZ4760frAzxe3gVwpRxgvaQyVPZE349~heFgGeZ-VFQX9h0tMe77s85tI80BQJvOpg8gFP2a3qsTRKXd74yM6wijOLVvMkXSbB0qWRV6GjaoLvujPDIqB3bgsTXN-CIWgc~W2BigLOtKGjJEcygApnzq53TSk5utP8xLTKGGfc8ZPg0UnNstHYp2RnZ04vYb~6hu24EAWz1bHZpKoK9~7adLdV3nsy-RBvIQzZuCOcq4ng__&Key-Pair-Id=APKAIE5G5CRDK6RD3PGA)
In vivo gene targeting of the FIX locus in FIX-KO mouse liver by AAV.SaCas9. Schematic diagrams of the mouse FIX locus show the SaCas9.sgRNA3 target site located in the 5′ end of exon 2; the AAV donor vector that contains U6-sgRNA3 and partial cDNA of codon-optimized, Padua-containing hFIX cDNA (hFIXco-Padua [exon 2-8]) followed by a polyA and flanked by homology arms; the modified mFIX locus after homologous recombination; the chimeric FIX mRNA transcribed from the gene-targeted FIX locus; and the translated chimeric FIX protein. Blue boxes indicate mFIX exons or exon-derived mRNA or protein sequences, and orange boxes indicate codon-optimized hFIX cDNA (exon 2-8), mRNA, or protein.