RNA 碱基编辑器:新出现的 RNA 治疗方法。

IF 6.4 2区 生物学 Q1 CELL BIOLOGY Wiley Interdisciplinary Reviews: RNA Pub Date : 2024-03-01 DOI:10.1002/wrna.1844
Jinghui Song, Nan Luo, Liting Dong, Jinying Peng, Chengqi Yi
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引用次数: 0

摘要

基于 RNA 的疗法为治疗遗传疾病提供了一种灵活、可逆的方法,如反义寡核苷酸、RNA 干扰、aptamers、mRNA 疫苗和 RNA 编辑。近年来,RNA 碱基编辑在纠正疾病相关点突变方面取得了重大进展。这些成就极大地影响了生物技术、生物医学研究和疗法开发领域。本文全面概述了当代 RNA 碱基编辑器的设计和性能,包括 A-to-I、C-to-U、A-to-m6A 和 U-to-Ψ。我们比较了最近的创新发展,并重点介绍了它们在疾病相关环境中的应用。最后,我们讨论了将 RNA 碱基编辑用于治疗目的的局限性和未来前景。本文归类于RNA 处理 > RNA 编辑和修饰 RNA 在疾病和发育中的应用 > RNA 在发育中的应用。
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RNA base editors: The emerging approach of RNA therapeutics.

RNA-based therapeutics offer a flexible and reversible approach for treating genetic disorders, such as antisense oligonucleotides, RNA interference, aptamers, mRNA vaccines, and RNA editing. In recent years, significant advancements have been made in RNA base editing to correct disease-relevant point mutations. These achievements have significantly influenced the fields of biotechnology, biomedical research and therapeutics development. In this article, we provide a comprehensive overview of the design and performance of contemporary RNA base editors, including A-to-I, C-to-U, A-to-m6A, and U-to-Ψ. We compare recent innovative developments and highlight their applications in disease-relevant contexts. Lastly, we discuss the limitations and future prospects of utilizing RNA base editing for therapeutic purposes. This article is categorized under: RNA Processing > RNA Editing and Modification RNA in Disease and Development > RNA in Development.

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来源期刊
CiteScore
14.80
自引率
4.10%
发文量
67
审稿时长
6-12 weeks
期刊介绍: WIREs RNA aims to provide comprehensive, up-to-date, and coherent coverage of this interesting and growing field, providing a framework for both RNA experts and interdisciplinary researchers to not only gain perspective in areas of RNA biology, but to generate new insights and applications as well. Major topics to be covered are: RNA Structure and Dynamics; RNA Evolution and Genomics; RNA-Based Catalysis; RNA Interactions with Proteins and Other Molecules; Translation; RNA Processing; RNA Export/Localization; RNA Turnover and Surveillance; Regulatory RNAs/RNAi/Riboswitches; RNA in Disease and Development; and RNA Methods.
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