作为治疗药物的小 RNA:从催化元件到调控途径。

IF 12.1 1区 医学 Q1 BIOTECHNOLOGY & APPLIED MICROBIOLOGY
Molecular Therapy Pub Date : 2025-05-07 Epub Date: 2025-04-02 DOI:10.1016/j.ymthe.2025.03.053
John J Rossi, Saumya Das
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引用次数: 0

摘要

RNA分子长期以来一直被认为在蛋白质合成中起着核心作用,主要是作为信使(mrna)、核糖体成分和接头(转移RNA)。然而,在过去的几十年里,具有调控或催化功能的小RNA的发现极大地扩展了我们对RNA生物学的理解。这些小rna可以针对特定转录物进行切割,改变mRNA翻译,直接基因启动子的表观遗传变化,甚至引导酶复合物到达其底物。在这篇综述中,我们强调并讨论了关键类型的小RNA的治疗潜力,包括核酶、RNA干扰元件、反义寡核苷酸、小核靶向RNA和转移RNA衍生的小RNA。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Small RNAs as therapeutic agents: From catalytic motifs to regulatory pathways.

RNA molecules have long been recognized for their central role in protein synthesis, primarily as messengers (mRNAs), ribosomal components, and adaptors (transfer RNAs). Over the past few decades, however, the discovery of small RNAs with regulatory or catalytic functions has dramatically expanded our understanding of RNA biology. These small RNAs can target specific transcripts for cleavage, alter mRNA translation, direct epigenetic changes at gene promoters, or even guide enzyme complexes to their substrates. In this review, we highlight and discuss the therapeutic potential of key classes of small RNAs, including ribozymes, RNA interference elements, antisense oligonucleotides, small nuclear-targeting RNAs, and transfer RNA-derived small RNAs.

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来源期刊
Molecular Therapy
Molecular Therapy 医学-生物工程与应用微生物
CiteScore
19.20
自引率
3.20%
发文量
357
审稿时长
3 months
期刊介绍: Molecular Therapy is the leading journal for research in gene transfer, vector development, stem cell manipulation, and therapeutic interventions. It covers a broad spectrum of topics including genetic and acquired disease correction, vaccine development, pre-clinical validation, safety/efficacy studies, and clinical trials. With a focus on advancing genetics, medicine, and biotechnology, Molecular Therapy publishes peer-reviewed research, reviews, and commentaries to showcase the latest advancements in the field. With an impressive impact factor of 12.4 in 2022, it continues to attract top-tier contributions.
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