Department of Chemical Engineering, Stanford University, Stanford, California 94305, United States.
Department of Bioengineering, Stanford University, Stanford, California 94305, United States.
ACS Synth Biol. 2024 Nov 15;13(11):3487-3496. doi: 10.1021/acssynbio.4c00183. Epub 2024 Oct 22.
Manipulating RNA species in mammalian cells has emerged as an important strategy for precise gene expression control. Here we review recent advances in precision transcriptome editing with a focus on tools that engineer specific transcripts for abundance, translation, base editing, alternative isoforms, and chemical modifications. While some of these methods have demonstrated efficiency in therapeutically relevant cellular or models, most require further study on their clinical safety and efficacy. Precision transcriptome engineering holds great potential for both mechanistic study of RNA biology and future gene and cell-based therapeutic applications.
在哺乳动物细胞中操纵 RNA 种类已成为精确基因表达控制的重要策略。在这里,我们综述了精确转录组编辑的最新进展,重点介绍了用于丰度、翻译、碱基编辑、替代异构体和化学修饰的特定转录本工程的工具。虽然这些方法中的一些在治疗相关的细胞或动物模型中显示出了效率,但大多数方法都需要进一步研究其临床安全性和有效性。精确转录组工程对于 RNA 生物学的机制研究和未来的基因和基于细胞的治疗应用具有巨大的潜力。