Genome Editing Redefines Precision Medicine in the Cardiovascular Field.

Elda Dzilic,Sean M Wu,Martina Dreßen,Stefanie A Doppler,Rüdiger Lange,Marcus-André Deutsch,Harald Lahm,Markus Krane

doi:10.1155/2018/4136473

Abstract

Genome editing is a powerful tool to study the function of specific genes and proteins important for development or disease. Recent technologies, especially CRISPR/Cas9 which is characterized by convenient handling and high precision, revolutionized the field of genome editing. Such tools have enormous potential for basic science as well as for regenerative medicine. Nevertheless, there are still several hurdles that have to be overcome, but patient-tailored therapies, termed precision medicine, seem to be within reach. In this review, we focus on the achievements and limitations of genome editing in the cardiovascular field. We explore different areas of cardiac research and highlight the most important developments: (1) the potential of genome editing in human pluripotent stem cells in basic research for disease modelling, drug screening, or reprogramming approaches and (2) the potential and remaining challenges of genome editing for regenerative therapies. Finally, we discuss social and ethical implications of these new technologies.

Highlights

The human genome project was a breakthrough for the scientific world
Especially CRISPR/Cas9 which is characterized by convenient handling and high precision, revolutionized the field of genome editing
We focus on the achievements and limitations of genome editing in the cardiovascular field

Summary

Introduction

The human genome project was a breakthrough for the scientific world. Knowing the sequence of the human genome allows the study of the function of specific genes or proteins [1]. The recently developed CRSIPR/Cas technology revolutionized the field of genome editing. A limiting factor, especially for the cardiovascular field since cardiomyocytes are largely postmitotic cells, is the fact that HDR is solely occurring in dividing cells, but first attempts are being developed to overcome this obstacle [6, 7]. This genome editing mechanism, the accuracy, and ease-of-use of the new technologies have enormous potential for basic science as well as for regenerative medicine. We discuss the social and ethical implications of these new technologies

Genome Editing in Basic Research

Regenerative Medicine in the Era of Genome

Main Challenge of Genome Editing

Ethical Concerns

Findings

Conclusion and Future Perspective