RNA therapeutics: Molecular mechanisms, and potential clinical translations.

3区 生物学 Q2 Biochemistry, Genetics and Molecular Biology
Tiep Tien Nguyen, Yen Vi Nguyen Thi, Dinh-Toi Chu
{"title":"RNA therapeutics: Molecular mechanisms, and potential clinical translations.","authors":"Tiep Tien Nguyen, Yen Vi Nguyen Thi, Dinh-Toi Chu","doi":"10.1016/bs.pmbts.2023.12.012","DOIUrl":null,"url":null,"abstract":"<p><p>RNA therapies involve the utilization of natural and artificial RNA molecules to control the expression and function of cellular genes and proteins. Initializing from 1990s, RNA therapies now show the rapid growth in the development and application of RNA therapeutics for treating various conditions, especially for undruggable diseases. The outstanding success of recent mRNA vaccines against COVID-19 infection again highlighted the important role of RNA therapies in future medicine. In this review, we will first briefly provide the crucial investigations on RNA therapy, from the first pieces of discovery on RNA molecules to clinical applications of RNA therapeutics. We will then classify the mechanisms of RNA therapeutics from various classes in the treatment of diseases. To emphasize the huge potential of RNA therapies, we also provide the key RNA products that have been on clinical trials or already FDA-approved. With comprehensive knowledge on RNA biology, and the advances in analysis, technology and computer-aid science, RNA therapies can bring a promise to be more expanding to the market in the future.</p>","PeriodicalId":49280,"journal":{"name":"Progress in Molecular Biology and Translational Science","volume":null,"pages":null},"PeriodicalIF":0.0000,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Progress in Molecular Biology and Translational Science","FirstCategoryId":"99","ListUrlMain":"https://doi.org/10.1016/bs.pmbts.2023.12.012","RegionNum":3,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2024/1/25 0:00:00","PubModel":"Epub","JCR":"Q2","JCRName":"Biochemistry, Genetics and Molecular Biology","Score":null,"Total":0}
引用次数: 0

Abstract

RNA therapies involve the utilization of natural and artificial RNA molecules to control the expression and function of cellular genes and proteins. Initializing from 1990s, RNA therapies now show the rapid growth in the development and application of RNA therapeutics for treating various conditions, especially for undruggable diseases. The outstanding success of recent mRNA vaccines against COVID-19 infection again highlighted the important role of RNA therapies in future medicine. In this review, we will first briefly provide the crucial investigations on RNA therapy, from the first pieces of discovery on RNA molecules to clinical applications of RNA therapeutics. We will then classify the mechanisms of RNA therapeutics from various classes in the treatment of diseases. To emphasize the huge potential of RNA therapies, we also provide the key RNA products that have been on clinical trials or already FDA-approved. With comprehensive knowledge on RNA biology, and the advances in analysis, technology and computer-aid science, RNA therapies can bring a promise to be more expanding to the market in the future.

RNA 疗法:分子机制和潜在的临床应用。
RNA 疗法涉及利用天然和人工 RNA 分子来控制细胞基因和蛋白质的表达和功能。RNA 疗法始于 20 世纪 90 年代,目前在治疗各种疾病,特别是无法用药物治疗的疾病方面,RNA 疗法的开发和应用呈现出快速增长的趋势。最近针对 COVID-19 感染的 mRNA 疫苗取得的巨大成功再次凸显了 RNA 疗法在未来医学中的重要作用。在这篇综述中,我们将首先简要介绍有关 RNA 疗法的重要研究,从 RNA 分子的首次发现到 RNA 疗法的临床应用。然后,我们将对各类 RNA 疗法治疗疾病的机制进行分类。为了强调 RNA 疗法的巨大潜力,我们还提供了已进入临床试验或已获得 FDA 批准的主要 RNA 产品。随着对 RNA 生物学知识的全面了解,以及分析、技术和计算机辅助科学的进步,RNA 疗法有望在未来扩大市场。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
求助全文
约1分钟内获得全文 求助全文
来源期刊
CiteScore
5.00
自引率
0.00%
发文量
110
审稿时长
4-8 weeks
期刊介绍: Progress in Molecular Biology and Translational Science (PMBTS) provides in-depth reviews on topics of exceptional scientific importance. If today you read an Article or Letter in Nature or a Research Article or Report in Science reporting findings of exceptional importance, you likely will find comprehensive coverage of that research area in a future PMBTS volume.
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
确定
请完成安全验证×
copy
已复制链接
快去分享给好友吧!
我知道了
右上角分享
点击右上角分享
0
联系我们:info@booksci.cn Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。 Copyright © 2023 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术官方微信