靶向RNA碱基编辑：技术演进、治疗应用及未来方向
关志宏 王刚（通讯作者）

广州大学生命科学学院精准基因编辑工程中心，广东广州，510006；

摘要：RNA碱基编辑技术作为校正疾病相关点突变的革命性工具，通过靶向系统引导效应蛋白实现RNA碱基的精准修饰，近年在效率与特异性上取得突破性进展。本文系统阐述RNA编辑系统的核心组成模块，重点综述尿苷到假尿苷（U→Ψ）、腺苷到N6-甲基腺苷（A→m6A）及N6-甲基腺苷到腺苷（m6A→A）编辑器的作用机制与研究进展，简要介绍递送策略，结合现状分析技术瓶颈并展望未来方向，为其临床转化提供参考。

关键词：RNA编辑；点突变；碱基修饰；靶向治疗；表观转录组

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