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. Author manuscript; available in PMC: 2017 Mar 1.
Published in final edited form as: Nature. 2016 Nov 16;540(7631):144–149. doi: 10.1038/nature20565

Figure 4. AAV-mediated systemic HITI in vivo.

Figure 4

a, Schematic of in vivo targeted GFP-NLS knock-in by HITI via intravenous (IV) AAV injections to Ai14 mice. b, Representative immunofluorescence images of GFP expression in the liver, heart, and quadriceps muscle after intravenous injection of HITI-AAVs (serotype 9). The absolute efficiencies of GFP knock-in per cell (liver and heart) and per nucleus (quadriceps muscle) were shown. Results were obtained from five sections per sample and presented as mean ± s.d. Scale bar, 200 μm. c, Validation of correct gene knock-in in heart and liver by genomic PCR. d, Frequencies of indel per target sequence of liver and heart by deep sequencing. e, Single-cell genotyping of GFP+ cells from HITI-AAVs injected Ai14 mouse muscle and heart. n, number of the analysed single cells. ND, not determined. For full gel image, see Supplementary Fig. 1. For source data, see Supplementary Table 4.