Luo Hui, Yao Jing, Zhang Rui
MOE Key Laboratory of Gene Function and Regulation, Guangdong Province Key Laboratory of Pharmaceutical Functional Genes, State Key Laboratory of Biocontrol, School of Life Sciences, Sun Yat-Sen University, Guangzhou, 510275, PR China.
Innovation Center for Evolutionary Synthetic Biology, School of Life Sciences, Sun Yat-Sen University, Guangzhou, 510275, PR China.
Adv Biotechnol (Singap). 2025 Apr 8;3(2):11. doi: 10.1007/s44307-025-00063-x.
Recent advancements in molecular engineering have established RNA-based technologies as powerful tools for both fundamental research and translational applications. Among the various RNA-based technologies developed, RNA base editing has recently emerged as a groundbreaking advancement. It primarily involves the conversion of adenosine (A) to inosine (I) and cytidine (C) to uridine (U), which are mediated by ADAR and APOBEC enzymes, respectively. RNA base editing has been applied in both biological research and therapeutic contexts. It enables site-directed editing within target transcripts, offering reversible, dose-dependent effects, in contrast to the permanent or heritable changes associated with DNA base editing. Additionally, RNA editing-based profiling of RNA-binding protein (RBP) binding sites facilitates transcriptome-wide mapping of RBP-RNA interactions in specific tissues and at the single-cell level. Furthermore, RNA editing-based sensors have been utilized to express effector proteins in response to specific RNA species. As RNA base editing technologies continue to evolve, we anticipate that they will significantly drive advancements in RNA therapeutics, synthetic biology, and biological research.
分子工程学的最新进展已将基于RNA的技术确立为基础研究和转化应用的强大工具。在已开发的各种基于RNA的技术中，RNA碱基编辑最近已成为一项突破性进展。它主要涉及分别由ADAR和APOBEC酶介导的腺苷（A）向肌苷（I）以及胞苷（C）向尿苷（U）的转化。RNA碱基编辑已应用于生物学研究和治疗领域。与DNA碱基编辑相关的永久性或遗传性变化不同，它能够在靶转录本内进行定点编辑，产生可逆的、剂量依赖性的效应。此外，基于RNA编辑的RNA结合蛋白（RBP）结合位点分析有助于在特定组织和单细胞水平上对RBP-RNA相互作用进行全转录组图谱绘制。此外，基于RNA编辑的传感器已被用于响应特定RNA种类表达效应蛋白。随着RNA碱基编辑技术不断发展，我们预计它们将显著推动RNA治疗、合成生物学和生物学研究的进展。
