CRISPR-Cas9 Genome Editing for Treatment of Atherogenic Dyslipidemia

Arterioscler Thromb Vasc Biol. 2018 Jan;38(1):12-18. doi: 10.1161/ATVBAHA.117.309326. Epub 2017 Aug 24.

Abstract

Although human genetics has resulted in the identification of novel lipid-related genes that can be targeted for the prevention of atherosclerotic vascular disease, medications targeting these genes or their protein products have short-term effects and require frequent administration during the course of the lifetime for maximal benefit. Genome-editing technologies, such as CRISPR-Cas9 (clustered regularly interspaced short palindromic repeats-CRISPR-associated 9) have the potential to permanently alter genes in the body and produce long-term and even lifelong protection against atherosclerosis. In this review, we discuss recent advances in genome-editing technologies and early proof-of-concept studies of somatic in vivo genome editing in mice that highlight the potential of genome editing to target disease-related genes in patients, which would establish a novel therapeutic paradigm for atherosclerosis.

Keywords: animals; atherosclerosis; gene editing; lipids; mice.

Publication types

  • Review

MeSH terms

  • Animals
  • Arteries / metabolism*
  • Arteries / pathology
  • Atherosclerosis / blood
  • Atherosclerosis / genetics
  • Atherosclerosis / pathology
  • Atherosclerosis / therapy*
  • CRISPR-Associated Proteins / genetics
  • CRISPR-Associated Proteins / metabolism*
  • CRISPR-Cas Systems*
  • Clustered Regularly Interspaced Short Palindromic Repeats*
  • Dyslipidemias / blood
  • Dyslipidemias / genetics
  • Dyslipidemias / pathology
  • Dyslipidemias / therapy*
  • Gene Editing / methods*
  • Gene Expression Regulation
  • Genetic Therapy / adverse effects
  • Genetic Therapy / methods*
  • Humans
  • Lipid Metabolism / genetics*
  • Lipids / blood*
  • Plaque, Atherosclerotic

Substances

  • CRISPR-Associated Proteins
  • Lipids