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=1725317648&Signature=bAUvhU~sDEASVavZCDtAb6634oo-c3lSBShH5wFQ66JN6Fos3oIAUUj0GMEtWPqjffZxy7BpEPEZQy6~GbkZBMUquunc-nBnbGB1nKltMXdQFHgsUi~lgSn9jzr3-EBMK3mHRn~rZxiquBd-SS6mXCtbJcxcpzrlYV3T7tJzmTUYN2KjgrPI7bK8lQnWllXm6zRSOGNbHbOdTvdqaMNE8dFq6RTdodyCXU6ip~nptQUqN~uBOIKLXr~ydYHREb0cWrfrHCNfS~l--yGEKRkrKgSw3Wa1Va79hIpMWKoqdgwy7MsPA4MJUkK9iVVLVXO34i50-8XNpPzpVum7KrTo7Q__&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.