Technologies for Targeted RNA Degradation and Induced RNA Decay

IF 51.4 1区 化学 Q1 CHEMISTRY, MULTIDISCIPLINARY Chemical Reviews Pub Date : 2024-11-05 DOI:10.1021/acs.chemrev.4c00472
Sigitas Mikutis, Gonçalo J. L. Bernardes
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Abstract

The vast majority of the human genome codes for RNA, but RNA-targeting therapeutics account for a small fraction of approved drugs. As such, there is great incentive to improve old and develop new approaches to RNA targeting. For many RNA targeting modalities, just binding is not sufficient to exert a therapeutic effect; thus, targeted RNA degradation and induced decay emerged as powerful approaches with a pronounced biological effect. This review covers the origins and advanced use cases of targeted RNA degrader technologies grouped by the nature of the targeting modality as well as by the mode of degradation. It covers both well-established methods and clinically successful platforms such as RNA interference, as well as emerging approaches such as recruitment of RNA quality control machinery, CRISPR, and direct targeted RNA degradation. We also share our thoughts on the biggest hurdles in this field, as well as possible ways to overcome them.

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靶向 RNA 降解和诱导 RNA 降解技术
人类基因组的绝大部分都是 RNA 编码,但 RNA 靶向疗法只占获批药物的一小部分。因此,改进旧的 RNA 靶向方法和开发新的 RNA 靶向方法有很大的动力。对于许多 RNA 靶向模式来说,仅仅结合还不足以产生治疗效果;因此,靶向 RNA 降解和诱导衰变成为具有显著生物效应的强大方法。本综述涵盖了靶向 RNA 降解技术的起源和先进应用案例,并按照靶向方式的性质和降解模式进行了分组。它既包括成熟的方法和临床上成功的平台(如 RNA 干扰),也包括新兴的方法(如招募 RNA 质量控制机制、CRISPR 和直接靶向 RNA 降解)。我们还分享了我们对这一领域最大障碍的看法,以及克服这些障碍的可能方法。
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来源期刊
Chemical Reviews
Chemical Reviews 化学-化学综合
CiteScore
106.00
自引率
1.10%
发文量
278
审稿时长
4.3 months
期刊介绍: Chemical Reviews is a highly regarded and highest-ranked journal covering the general topic of chemistry. Its mission is to provide comprehensive, authoritative, critical, and readable reviews of important recent research in organic, inorganic, physical, analytical, theoretical, and biological chemistry. Since 1985, Chemical Reviews has also published periodic thematic issues that focus on a single theme or direction of emerging research.
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