Gene editing therapy for cardiovascular diseases

Abstract

The development of gene editing tools has been a significant area of research in the life sciences for nearly 30 years. These tools have been widely utilized in disease detection and mechanism research. In the new century, they have shown potential in addressing various scientific challenges and saving lives through gene editing therapies, particularly in combating cardiovascular disease (CVD). The rapid advancement of gene editing therapies has provided optimism for CVD patients. The progress of gene editing therapy for CVDs is a comprehensive reflection of the practical implementation of gene editing technology in both clinical and basic research settings, as well as the steady advancement of research and treatment of CVDs. This article provides an overview of the commonly utilized DNA-targeted gene editing tools developed thus far, with a specific focus on the application of these tools, particularly the clustered regularly interspaced short palindromic repeat/CRISPR-associated genes (Cas) (CRISPR/Cas) system, in CVD gene editing therapy. It also delves into the challenges and limitations of current gene editing therapies, while summarizing ongoing research and clinical trials related to CVD. The aim is to facilitate further exploration by relevant researchers by summarizing the successful applications of gene editing tools in the field of CVD.

Keywords: CRISPR/Cas; cardiovascular disease; gene therapy; lipid nanoparticles.

FIGURE 1

The discovery and development of the CRISPR/Cas system. The CRISPR system has rapidly expanded since its initial discovery, emerging as a crucial tool for gene editing therapies. Abbreviations: ABE, Adenine Base Editor; CBE, Cytidine Base Editor; CRISPR, clustered regularly interspaced short palindromic repeats; Cas, CRISPR‐associated.

FIGURE 2

The application of the CRISPR/Cas system for gene editing therapy in CVD. A variety of gene editing systems centered on the CRISPR/Cas system have demonstrated effective gene editing outcomes in various animal models targeting key genes of CVDs with different delivery methods, and furthermore have achieved initial successes in gene editing therapies for CVDs in human clinics. Abbreviations: DMD, dystrophin; LDL‐C, low‐density lipoprotein cholesterol; TG, triglyceride; TTR, transthyretin.