Figure 3.

Genome Editing Activity in Human iPSCs and in the Heart of an EGFP Mouse Model

(A) VEsiCas activity in induced pluripotent stem cells (iPSCs). Shown is the percentage of non-fluorescent iPSCs stably expressing EGFP after treatment with VEsiCas carrying either sgCtr or sgGFPI2. Data are presented as mean ± SEM for n = 2 independent experiments. (B) VEsiCas-mediated gene disruption in the cardiac muscle of EGFP mice. Shown are fluorescence microscopy images of cardiac tissue sections from EGFP transgenic mice 10 days after intra-cardiac injection of VEsiCas carrying sgCtr or sgEGFPBi. DAPI was used as a nuclear counterstain. Immunostaining for α-actinin was performed to identify cardiomyocytes (bottom). A representative sample of n = 5 experiments is shown. Scale bar, 100 μm. (C) Quantification of VEsiCas EGFP knockout in cardiac tissue. The bar graph shows the percentage of non-fluorescent cardiomyocytes following VEsiCas treatments (mean ± SEM of n = 5 independent experiments).