Genome Editing for Cardiovascular Diseases—A Brief Review for Cardiologists

Abstract

The recent technical advances in genome engineering have accelerated our understanding of the molecular mechanisms of human diseases and are leading to increased clinical applications of gene-targeting therapies. The field of cardiovascular medicine, rich in knowledge of molecular level disease mechanisms, is particularly well positioned to receive significant benefits from this technology. Specifically, a new generation of genome editing tools capable of introducing targeted sequence modifications at high frequencies initiated by induced DNA double-strand breaks has been developed. Of note is the RNA-guided genome editing system, clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR associated protein 9 (Cas9), which has provided researchers and clinicians a malleable gene-targeting platform with high specificity. Recent reports have robustly demonstrated proof-of-concept in using CRISPR-Cas9 based gene therapy for treating common cardiovascular diseases and are testaments that the new genome editing technology holds promise for treating patients with cardiovascular ailments in the clinic in the near future. In light of this trend, a basic understanding of genome editing technology is becoming more relevant to clinical cardiologists. To this end, a concise explanation of terms and the biological basis of genome editing, on-going research, and clinical trials highly relevant to clinical application are presented. In conclusion, the aim of this short review is to introduce clinicians to the core concepts of current genome editing technology.