Integrating Prime Editing and Cellular Reprogramming as Novel Strategies for Genetic Cardiac Disease Modeling and Treatment

Curr Cardiol Rep. 2024 Nov;26(11):1197-1208. doi: 10.1007/s11886-024-02118-2. Epub 2024 Sep 11.

Abstract

Purpose of review: This review aims to evaluate the potential of CRISPR-based gene editing tools, particularly prime editors (PE), in treating genetic cardiac diseases. It seeks to answer how these tools can overcome current therapeutic limitations and explore the synergy between PE and induced pluripotent stem cell-derived cardiomyocytes (iPSC-CMs) for personalized medicine.

Recent findings: Recent advancements in CRISPR technology, including CRISPR-Cas9, base editors, and PE, have demonstrated precise genome correction capabilities. Notably, PE has shown exceptional precision in correcting genetic mutations. Combining PE with iPSC-CMs has emerged as a robust platform for disease modeling and developing innovative treatments for genetic cardiac diseases. The review finds that PE, when combined with iPSC-CMs, holds significant promise for treating genetic cardiac diseases by addressing their root causes. This approach could revolutionize personalized medicine, offering more effective and precise treatments. Future research should focus on refining these technologies and their clinical applications.

Keywords: CRISPR-based gene editing; Cellular reprogramming; Gene therapy; Genetic cardiac diseases; Prime editing.

Publication types

  • Review

MeSH terms

  • CRISPR-Cas Systems*
  • Cellular Reprogramming*
  • Gene Editing* / methods
  • Genetic Therapy* / methods
  • Heart Diseases* / genetics
  • Heart Diseases* / therapy
  • Humans
  • Induced Pluripotent Stem Cells*
  • Myocytes, Cardiac*
  • Precision Medicine