Innovations in RNA therapeutics: a review of recent advances and emerging technologies.

IF 1.1 4区生物学 Q4 BIOCHEMISTRY & MOLECULAR BIOLOGY Nucleosides, Nucleotides & Nucleic Acids Pub Date : 2025-01-13 DOI:10.1080/15257770.2025.2451377

Tuward J Dweh, Glay Jr Eric Wulu, John Kessellie Jallah, Dominic L Miller, Jyoti Prakash Sahoo

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Abstract

The field of biomedical science has witnessed another milestone with the advent of RNA-based therapeutics. This review explores three major RNA molecules, namely: messenger RNA (mRNA), RNA interference technology (RNAi), and Antisense Oligonucleotide (ASO), and analyses U.S. Food and Drug Administration drugs from 14 RNA-based pharmaceutical companies in terms of targeted genes, diseases and types, clinical trials and status, the mode of delivery, and the year of production. Many of such drugs are clinically approved or pending approval by the U.S. Food and Drug Administration (FDA) alongside other leading drugs agencies. Regarding diseases, this article emphasizes cancer therapy, genetic diseases, viral infections, and two categories of drug delivery systems include viral vectors and nanoparticles. Despite the tremendous progress made, key issues associated with these delivery systems are stability, off-target activities of RNA payloads, efficiency in cellular uptake, and the innovative need for engineering techniques for modifications. This review emphasizes the transformative potential of RNA therapeutics and the role of innovative technologies in addressing clinical needs, paving the way for a new era in precision medicine.

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RNA疗法的创新：最新进展和新兴技术综述。

随着rna疗法的出现，生物医学科学领域见证了另一个里程碑。本文综述了信使RNA （mRNA）、RNA干扰技术（RNAi）和反义寡核苷酸（ASO）这三种主要RNA分子，并从靶向基因、疾病和类型、临床试验和现状、给药方式和生产年份等方面分析了美国食品和药物管理局（fda） 14家基于RNA的制药公司的药物。许多此类药物已获得美国食品和药物管理局（FDA）以及其他主要药物机构的临床批准或等待批准。在疾病方面，本文重点介绍了癌症治疗、遗传病、病毒感染，以及病毒载体和纳米颗粒两类药物传递系统。尽管取得了巨大的进展，但与这些传递系统相关的关键问题是稳定性、RNA有效载荷的脱靶活性、细胞摄取的效率以及对工程修饰技术的创新需求。这篇综述强调了RNA疗法的变革潜力和创新技术在解决临床需求方面的作用，为精准医学的新时代铺平了道路。

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

Nucleosides, Nucleotides & Nucleic Acids 生物-生化与分子生物学

CiteScore

2.60

自引率

7.70%

发文量

审稿时长

6 months

期刊介绍： Nucleosides, Nucleotides & Nucleic Acids publishes research articles, short notices, and concise, critical reviews of related topics that focus on the chemistry and biology of nucleosides, nucleotides, and nucleic acids. Complete with experimental details, this all-inclusive journal emphasizes the synthesis, biological activities, new and improved synthetic methods, and significant observations related to new compounds.