Ablation of CaMKIIδ oxidation by CRISPR-Cas9 base editing as a therapy for cardiac disease

Science. 2023 Jan 13;379(6628):179-185. doi: 10.1126/science.ade1105. Epub 2023 Jan 12.

Abstract

CRISPR-Cas9 gene editing is emerging as a prospective therapy for genomic mutations. However, current editing approaches are directed primarily toward relatively small cohorts of patients with specific mutations. Here, we describe a cardioprotective strategy potentially applicable to a broad range of patients with heart disease. We used base editing to ablate the oxidative activation sites of CaMKIIδ, a primary driver of cardiac disease. We show in cardiomyocytes derived from human induced pluripotent stem cells that editing the CaMKIIδ gene to eliminate oxidation-sensitive methionine residues confers protection from ischemia/reperfusion (IR) injury. Moreover, CaMKIIδ editing in mice at the time of IR enables the heart to recover function from otherwise severe damage. CaMKIIδ gene editing may thus represent a permanent and advanced strategy for heart disease therapy.

MeSH terms

  • Animals
  • CRISPR-Cas Systems
  • Calcium-Calmodulin-Dependent Protein Kinase Type 2* / genetics
  • Gene Editing*
  • Heart Diseases* / genetics
  • Heart Diseases* / therapy
  • Humans
  • Induced Pluripotent Stem Cells / enzymology
  • Mice
  • Myocytes, Cardiac / enzymology

Substances

  • Calcium-Calmodulin-Dependent Protein Kinase Type 2