Genome Editing in Dyslipidemia and Atherosclerosis

Adv Exp Med Biol. 2023:1396:139-156. doi: 10.1007/978-981-19-5642-3_10.

Abstract

Despite successive advancement of genome editing technology with zinc finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs), the recent breakthrough in the field has been related to clustered regularly interspaced short palindromic repeats/associated proteins (CRISPR/Cas). The high efficiency and convenience of CRIPSR/Cas systems dramatically accelerate pre- and clinical experimentations of dyslipidemia and atherosclerosis. In this chapter, we review the latest state of genome editing in translational research of dyslipidemia and atherosclerosis. We highlight recent progress in therapeutic development for familial dyslipidemia by genome editing. We point to the challenges in maximizing efficacy and minimizing safety issues related to the once-and-done therapy focusing on CRISPR/Cas systems. We give an outlook on the potential gene targets prioritized by large-scale genetic studies of cardiovascular diseases and genome editing in precision medicine of dyslipidemia and atherosclerosis.

Keywords: Atherosclerosis; CRISPR/Cas; Dyslipidemia; Familial hypercholesterolemia; Genome editing; Genome-wide association study; Precision medicine.

Publication types

  • Review

MeSH terms

  • Atherosclerosis* / genetics
  • Atherosclerosis* / therapy
  • Cardiovascular Diseases*
  • Dyslipidemias* / genetics
  • Dyslipidemias* / therapy
  • Gene Editing
  • Humans
  • Transcription Activator-Like Effector Nucleases

Substances

  • Transcription Activator-Like Effector Nucleases