Genome editing in cardiovascular diseases

Prog Mol Biol Transl Sci. 2021:181:289-308. doi: 10.1016/bs.pmbts.2021.01.021. Epub 2021 Feb 22.

Abstract

Genetic modification at the molecular level in somatic cells, germline, and animal models requires for different purposes, such as introducing desired mutation, deletion of alleles, and insertion of novel genes in the genome. Various genome-editing tools are available to accomplish these alterations, such as zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR associated (Cas) system. CRISPR-Cas system is an emerging technology, which is being used in biological and medical sciences, including in the cardiovascular field. It assists to identify the mechanism of various cardiovascular disease occurrence, such as hypertrophic cardiomyopathy (HCM), dilated cardiomyopathy (DCM), and arrhythmogenic cardiomyopathy (ACM). Furthermore, it has been advantages to edit various genes simultaneously and can also be used to treat and prevent several human diseases. This chapter explores the use of the scientific and therapeutic potential of a CRISPR-Cas system to edit the various cardiovascular disease-associated genes to understand the pathways involved in disease progression and treatment.

Keywords: CRISPR-Cas9; Cardiac; Cardiovascular disease; Genome editing; Heart failure; iPSC.

MeSH terms

  • Animals
  • CRISPR-Cas Systems / genetics
  • Cardiovascular Diseases* / genetics
  • Cardiovascular Diseases* / therapy
  • Gene Editing*
  • Humans
  • Transcription Activator-Like Effector Nucleases / metabolism
  • Zinc Finger Nucleases

Substances

  • Transcription Activator-Like Effector Nucleases
  • Zinc Finger Nucleases