Genome Editing and Pathological Cardiac Hypertrophy

Adv Exp Med Biol. 2023:1396:87-101. doi: 10.1007/978-981-19-5642-3_6.

Abstract

Three major genome editing tools, transcription activator-like effector nucleases (TALENs), zinc finger nucleases (ZFNs), and clustered regularly interspaced short palindromic repeat (CRISPR) systems, are increasingly important technologies used in the study and treatment of hereditary myocardial diseases. Germ cell genome editing and modification can permanently eliminate monogenic cardiovascular disease from the offspring of affected families and the next generation, although ethically controversial. Somatic genome editing may be a promising method for the treatment of hereditary cardiomyopathy various diseases for which gene knockout is favorable and can also treat people who are already ill, although there are currently some technical challenges. This chapter describes the application of genome editing in the experimental studies and treatment of hypertrophic cardiomyopathy as well as other cardiomyopathies.

Keywords: CRISPR/Cas; DMD; Genome editing; HCM.

MeSH terms

  • Cardiomegaly / genetics
  • Cardiomegaly / therapy
  • Cardiomyopathy, Hypertrophic*
  • Clustered Regularly Interspaced Short Palindromic Repeats / genetics
  • Gene Editing*
  • Humans
  • Transcription Activator-Like Effector Nucleases / genetics

Substances

  • Transcription Activator-Like Effector Nucleases