Therapeutic application of genome editing in dyslipidemia

Curr Opin Lipidol. 2022 Apr 1;33(2):133-138. doi: 10.1097/MOL.0000000000000805.

Abstract

Purpose of review: To summarize recent advances with respect to the use of genome editing to modify blood lipid levels in vivo.

Recent findings: Genome-editing technologies have been successfully used to target the PCSK9 gene in the livers of nonhuman primates and significantly reduce blood LDL cholesterol levels.

Summary: Multiple proof-of-concept nonhuman primate studies raise the prospect of genome editing empowering 'one-and-done' therapies for the treatment of dyslipidemic patients.

Publication types

  • Review

MeSH terms

  • Animals
  • Dyslipidemias* / genetics
  • Dyslipidemias* / therapy
  • Gene Editing* / methods
  • Humans
  • Liver
  • Proprotein Convertase 9 / genetics

Substances

  • PCSK9 protein, human
  • Proprotein Convertase 9