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. Author manuscript; available in PMC: 2015 Oct 9.
Published in final edited form as: Nature. 2015 Apr 1;520(7546):186–191. doi: 10.1038/nature14299

Figure 4. AAV-delivery of SaCas9 for in vivo genome editing.

Figure 4

a, Single-vector AAV system and experimental timeline. b, Indels at Pcsk9 targets in liver tissue following injection of AAV at 2E11 total genome copies (n=3 animals). Time course of c, serum Pcsk9 and d, total cholesterol in animals (n=3 for all titers and time points, error bars show S.E.M.). e, Manhattan plots of BLESS-identified DSB clusters in N2a cells. Inset indicates indel levels at top DSB scoring loci. f, Indels in liver tissue (n=3 animals, error bars indicate Wilson intervals) at BLESS-identified off-target loci. Heatmap indicates DSB scores.