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. 2017 Mar 3;120(5):778-780.
doi: 10.1161/CIRCRESAHA.116.310197.

Genome Editing in Cardiovascular Biology

Timon Seeger  1 , Matthew Porteus  1 , Joseph C Wu  2
Affiliations

Genome Editing in Cardiovascular Biology

Timon Seeger et al. Circ Res. .

Abstract

Genome editing has emerged as a powerful tool in research and is entering the stage of therapeutic applications. In the cardiovascular field, its role in basic and translational research is well established. However, biological and technical barriers currently hamper the therapeutic potential of genome editing for cardiovascular diseases. This viewpoint discusses possible routes for promoting therapeutic use of genome editing in the cardiovascular system.

Keywords: CRISPR; cardiomyopathies; cardiovascular diseases; gene editing; induced pluripotent stem cells.

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Figures

Figure 1
Figure 1. Genome editing in cardiovascular development, biology, and therapy
a) The main programmable nucleases are ZFN, TALEN and CRISPR. b) They induce DSBs at specific DNA loci leading to NHEJ as well as HDR. Modified nucleases can be employed for transcription interference without causing DSB. Recent advances in CRISPR technology also allows for targeting RNA. c) Genome editing already facilitates the generation of genetically modified animals to study cardiovascular development and biology and plays a major role in disease modeling. However, the role of genome editing in therapeutic approaches for cardiovascular diseases is less evident. Ex vivo and in vivo approaches are possible routes for therapeutic applications. Autologous iPSCs can be modified ex vivo and differentiated into the desired cell type for cell therapy. Further, mutations might be directly targeted in vivo or excised by exon skipping. (ZFN, zinc finger nuclease; DSB, double strand break; NHEJ, non-homologous end joining; HDR, homologous directed repair; c-iPSC, corrected iPSC; CM, cardiomyocyte; EC, endothelial cell).
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