Current RNA strategies in treating cardiovascular diseases

Mol Ther. 2024 Mar 6;32(3):580-608. doi: 10.1016/j.ymthe.2024.01.028. Epub 2024 Jan 29.

Abstract

Cardiovascular disease (CVD) continues to impose a significant global health burden, necessitating the exploration of innovative treatment strategies. Ribonucleic acid (RNA)-based therapeutics have emerged as a promising avenue to address the complex molecular mechanisms underlying CVD pathogenesis. We present a comprehensive review of the current state of RNA therapeutics in the context of CVD, focusing on the diverse modalities that bring about transient or permanent modifications by targeting the different stages of the molecular biology central dogma. Considering the immense potential of RNA therapeutics, we have identified common gene targets that could serve as potential interventions for prevalent Mendelian CVD caused by single gene mutations, as well as acquired CVDs developed over time due to various factors. These gene targets offer opportunities to develop RNA-based treatments tailored to specific genetic and molecular pathways, presenting a novel and precise approach to address the complex pathogenesis of both types of cardiovascular conditions. Additionally, we discuss the challenges and opportunities associated with delivery strategies to achieve targeted delivery of RNA therapeutics to the cardiovascular system. This review highlights the immense potential of RNA-based interventions as a novel and precise approach to combat CVD, paving the way for future advancements in cardiovascular therapeutics.

Keywords: CRISPR; RNA interference; RNA therapeutics; adeno-associated virus; antisense oligonucleotide; cardiovascular disease; genetic variants; lipid nanoparticle; therapeutic delivery.

Publication types

  • Review

MeSH terms

  • Cardiovascular Diseases* / genetics
  • Cardiovascular Diseases* / therapy
  • Humans
  • MicroRNAs*
  • RNA

Substances

  • RNA
  • MicroRNAs