Utilization and Potential of RNA-Based Therapies in Cardiovascular Disease

Emma Louise Robinson; J David Port

doi:10.1016/j.jacbts.2022.02.003

Utilization and Potential of RNA-Based Therapies in Cardiovascular Disease

JACC Basic Transl Sci. 2022 Jun 8;7(9):956-969. doi: 10.1016/j.jacbts.2022.02.003. eCollection 2022 Sep.

Authors

Emma Louise Robinson^{1

2}, J David Port^{2

3}

Affiliations

¹ Consortium for Fibrosis Research and Translation (CFReT), University of Colorado, Anschutz Medical Campus, Aurora, Colorado, USA.
² Department of Medicine, Division of Cardiology, University of Colorado School of Medicine, Anschutz Medical Campus, Denver, Colorado, USA.
³ Department of Pharmacology, University of Colorado School of Medicine, Anschutz Medical Campus, Denver, Colorado, USA.

Abstract

Cardiovascular disease (CVD) remains the largest cause of mortality worldwide. The development of new effective therapeutics is a major unmet need. The current review focuses broadly on the concept of nucleic acid (NA)-based therapies, considering the use of various forms of NAs, including mRNAs, miRNAs, siRNA, and guide RNAs, the latter specifically for the purpose of CRISPR-Cas directed gene editing. We describe the current state-of-the-art of RNA target discovery and development, the status of RNA therapeutics in the context of CVD, and some of the challenges and hurdles to be overcome.

Keywords: AAV, adeno-associated viral vector; ASO, antisense oligonucleotide; CRISPR, clustered regularly interspaced short palindromic repeats; CVD, cardiovascular disease; DMD, Duchenne muscular dystrophy; HF, heart failure; NA, nucleic acid; RNA; cardiovascular disease; circRNA, circular RNA; lncRNA, long noncoding RNA; miRNA, microRNA; ncRNA, noncoding RNA; noncoding RNA; nucleic acid-based therapies; therapeutic modalities.

Publication types

Review