将 Prime 编辑与细胞重编程相结合，作为遗传心脏疾病建模和治疗的新策略。

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

机构信息

Experimental Cardiology Laboratory, Department of Cardiology, Division of Heart and Lungs, University Medical Center Utrecht, Utrecht, The Netherlands.

Regenerative Medicine Center Utrecht, Circulatory Health Research Center, University Medical Center Utrecht, University Utrecht, Utrecht, The Netherlands.

出版信息

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

DOI:10.1007/s11886-024-02118-2

PMID:39259489

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11538137/

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.

摘要

目的综述：本综述旨在评估基于 CRISPR 的基因编辑工具，特别是 Prime 编辑器（PE），在治疗遗传性心脏疾病方面的潜力。它试图回答这些工具如何克服当前的治疗局限性，并探讨 PE 与诱导多能干细胞衍生的心肌细胞（iPSC-CMs）在个性化医疗方面的协同作用。

最近发现：CRISPR 技术的最新进展，包括 CRISPR-Cas9、碱基编辑器和 PE，已经展示了精确的基因组校正能力。值得注意的是，PE 在纠正遗传突变方面表现出了非凡的精确性。PE 与 iPSC-CMs 的结合已经成为疾病建模和开发遗传性心脏疾病创新治疗方法的强大平台。本综述发现，PE 与 iPSC-CMs 结合，通过解决遗传性心脏疾病的根本原因，为治疗此类疾病带来了巨大的希望。这种方法可能会彻底改变个性化医疗，提供更有效和精确的治疗方法。未来的研究应集中于改进这些技术及其临床应用。