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. 2020 Feb 12;6(7):eaax5701. doi: 10.1126/sciadv.aax5701

Fig. 5. Time course of OTC expression in liver by neonatal gene therapy, CRISPR-Cas9–mediated gene correction, or gene targeting.

Fig. 5

p2 spfash mice received temporal vein injection of AAV8.SaCas9 (5 × 1010 GC per pup) and 5 × 1011 GC per pup of AAV8.sRNA1.donor vector for CRISPR-Cas9–mediated gene correction (A), AAV8.control.hOTCco donor (equivalent of a gene therapy vector, B), or AAV8.sgRNA1.hOTCco donor for CRISPR-Cas9–mediated gene targeting (C). Liver samples were harvested at 1, 3, 7, 21, and 56 days after vector injection for immunostaining with an OTC antibody. Representative pictures at each time point are shown. Scale bar, 200 μm.