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用于表观基因组的精密手术刀:靶向治疗中的新一代编辑工具。

Precision scalpels for the epigenome: next-gen editing tools in targeted therapies.

作者信息

Zhang Ruiming, Yao Tianqi, Fan Meiyin, Jiang Xiaoying, Wang Keshan, Cui Min, Bing Kaijian, Xia Xiaotian

机构信息

Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China.

Health Management Center, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China.

出版信息

Front Med (Lausanne). 2025 Jun 17;12:1613722. doi: 10.3389/fmed.2025.1613722. eCollection 2025.

Abstract

This article reviews the fundamental concepts of epigenetics and its related mechanisms, and discusses recent advances in epigenetic gene editors and their applications in disease treatment. First, the article introduces the concept of epigenetic inheritance and the four main epigenetic mechanisms. Then, after briefly outlining traditional gene editing, it presents epigenetic gene editors, their associated tools, and the historical context of their development. Subsequently, the article describes the working principles and advantages of epigenetic editing tools such as KRAB and DNMT. Addressing the current bottlenecks in the field, the article provides an in-depth analysis of editing efficiency and specificity, long-term safety, and the complexity of clinical applications. In addition, it discusses optimization strategies for delivery systems, minimization of off-target effects, and therapeutic approaches for multigene disorders. Finally, the article outlines the progress of epigenetic editors in both neoplastic and non-neoplastic disease research. In summary, this article offers a comprehensive review of the theoretical foundations of epigenetics, the evolution of gene editing tools, and the latest advances in epigenetic editors for disease treatment, providing a valuable reference for future research and clinical application.

摘要

本文回顾了表观遗传学的基本概念及其相关机制,并讨论了表观遗传基因编辑器的最新进展及其在疾病治疗中的应用。首先,文章介绍了表观遗传遗传的概念和四种主要的表观遗传机制。然后,在简要概述传统基因编辑后,介绍了表观遗传基因编辑器、其相关工具及其发展的历史背景。随后,文章描述了诸如KRAB和DNMT等表观遗传编辑工具的工作原理和优势。针对该领域当前的瓶颈,文章对编辑效率和特异性、长期安全性以及临床应用的复杂性进行了深入分析。此外,还讨论了递送系统的优化策略、脱靶效应的最小化以及多基因疾病的治疗方法。最后,文章概述了表观遗传编辑器在肿瘤和非肿瘤疾病研究中的进展。总之,本文对表观遗传学的理论基础、基因编辑工具的演变以及用于疾病治疗的表观遗传编辑器的最新进展进行了全面综述,为未来的研究和临床应用提供了有价值的参考。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/17f8/12209235/cb2e6e8caae8/fmed-12-1613722-g001.jpg

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