CRISPR and cardiovascular diseases

Cardiovasc Res. 2023 Mar 17;119(1):79-93. doi: 10.1093/cvr/cvac048.

Abstract

CRISPR technologies have progressed by leaps and bounds over the past decade, not only having a transformative effect on biomedical research but also yielding new therapies that are poised to enter the clinic. In this review, I give an overview of (i) the various CRISPR DNA-editing technologies, including standard nuclease gene editing, base editing, prime editing, and epigenome editing, (ii) their impact on cardiovascular basic science research, including animal models, human pluripotent stem cell models, and functional screens, and (iii) emerging therapeutic applications for patients with cardiovascular diseases, focusing on the examples of hypercholesterolaemia, transthyretin amyloidosis, and Duchenne muscular dystrophy.

Keywords: CRISPR; base editing; cardiovascular disease; gene editing; genome editing.

Publication types

  • Review

MeSH terms

  • Animals
  • CRISPR-Cas Systems
  • Cardiovascular Diseases* / diagnosis
  • Cardiovascular Diseases* / genetics
  • Cardiovascular Diseases* / therapy
  • Clustered Regularly Interspaced Short Palindromic Repeats
  • Gene Editing
  • Genetic Therapy
  • Humans
  • Muscular Dystrophy, Duchenne* / genetics