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

IF 3.1 3区医学 Q2 CARDIAC & CARDIOVASCULAR SYSTEMS Current Cardiology Reports Pub Date : 2024-09-11 DOI:10.1007/s11886-024-02118-2

Bing Yao, Zhiyong Lei, Manuel A. F. V. Gonçalves, Joost P. G. Sluijter

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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.

Summary

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.

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整合基因编辑和细胞重编程作为遗传性心脏病建模和治疗的新策略

综述目的本综述旨在评估基于CRISPR的基因编辑工具，尤其是素体编辑器（PE）在治疗遗传性心脏疾病方面的潜力。最近的研究结果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 在纠正基因突变方面表现出了非凡的精确性。综述发现，PE 与 iPSC-CMs 结合后，有望从根本上治疗遗传性心脏疾病。这种方法可彻底改变个性化医学，提供更有效、更精确的治疗。未来的研究应侧重于完善这些技术及其临床应用。

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来源期刊

Current Cardiology Reports CARDIAC & CARDIOVASCULAR SYSTEMS-

CiteScore

6.20

自引率

2.70%

发文量

209

期刊介绍： The aim of this journal is to provide timely perspectives from experts on current advances in cardiovascular medicine. We also seek to provide reviews that highlight the most important recently published papers selected from the wealth of available cardiovascular literature. We accomplish this aim by appointing key authorities in major subject areas across the discipline. Section editors select topics to be reviewed by leading experts who emphasize recent developments and highlight important papers published over the past year. An Editorial Board of internationally diverse members suggests topics of special interest to their country/region and ensures that topics are current and include emerging research. We also provide commentaries from well-known figures in the field.