RNA therapeutics: Molecular mechanisms, and potential clinical translations.

3区生物学 Q2 Biochemistry, Genetics and Molecular Biology

Progress in Molecular Biology and Translational Science Pub Date : 2024-01-01 Epub Date: 2024-01-25 DOI:10.1016/bs.pmbts.2023.12.012

Tiep Tien Nguyen, Yen Vi Nguyen Thi, Dinh-Toi Chu

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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.

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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。

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来源期刊

Progress in Molecular Biology and Translational Science BIOCHEMISTRY & MOLECULAR BIOLOGY-

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.