A glance at the application of CRISPR/Cas9 gene-editing technology in cardiovascular diseases

Abstract

Cardiovascular diseases (CVDs) remain major causes of global mortality in the world. Genetic approaches have succeeded in discovery of the molecular basis of an increasing number of cardiac diseases. Genome editing strategies are one of the most effective methods for assisting therapeutic approaches. Potential therapeutic methods of correcting disease-causing mutations or of knocking out specific genes as approaches for the prevention of CVDs have gained substantial attention using genome editing techniques. Recently, the clustered regularly interspaced short palindromic repeats/CRISPR-associated protein 9 (CRISPR/Cas9) system has become the most widely used genome-editing technology in molecular biology due to its benefits such as simple design, high efficiency, good repeatability, short-cycle, and costeffectiveness. In the present review, we discuss on the possibilities of applying the CRISPR/Cas9 genome editing tool in the CVDs.

Genome Editing and Cardiac Arrhythmias

“…The first publication of somatic genome editing of the heart involved using this system to correct Duchenne's muscular dystrophy (DMD) in a mouse model [15]. Subsequently, there have been several developments in expanding the capabilities and efficacy of the CRISPR/Cas9 systems and these are reviewed in the subsequent sections [16].…”

“…1 (14)(15)(16)(17)(18)(19) to minimize wild-type allele cleavage. The Cas9 endonuclease introduces a double-stranded break (DSB).…”

“…Schematic overview of major CRISPR/Cas9-based genome editing technologies. (A) Conventional CRISPR/Cas9 editing of a mutant allele involves selecting guide RNAs adjacent to a protospacer adjacent motif (PAM) site, with a mutation in the spacer sequence(14)(15)(16)(17)(18)(19) to minimize wild-type allele cleavage. The Cas9 endonuclease introduces a double-stranded break (DSB).…”