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. Author manuscript; available in PMC: 2016 Aug 1.
Published in final edited form as: Nat Biotechnol. 2016 Feb 1;34(3):334–338. doi: 10.1038/nbt.3469

Figure 1. In vivo gene correction of the OTC locus in the spfash mouse liver by AAV.CRISPR-SaCas9.

Figure 1

(a) Schematic diagram of the mouse OTC locus showing the spfash mutation and three SaCas9 targets. spfash has a G-to-A mutation at the donor splice site at the end of exon 4 indicated in red on the top strand. The three selected SaCas9-targeted genomic sites (20 bp each) are in blue and underlined with the PAM sequences marked in green. The black line above exon 4 indicates the 1.8 kb OTC donor template. (b) Dual AAV vector system for liver-directed and SaCas9-mediated gene correction. The AAV8.sgRNA1.donor vector contains a 1.8-kb murine OTC donor template sequence as shown in (a) with the corresponding PAM sequence mutated and an AgeI site inserted. (c) Flowchart showing the key steps of AAV8.CRISPR-SaCas9-mediated gene correction in the neonatal OTC spfash model.