{"title":"作为治疗药物的小 RNA:从催化元件到调控途径。","authors":"John J Rossi, Saumya Das","doi":"10.1016/j.ymthe.2025.03.053","DOIUrl":null,"url":null,"abstract":"<p><p>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.</p>","PeriodicalId":19020,"journal":{"name":"Molecular Therapy","volume":" ","pages":"2238-2242"},"PeriodicalIF":12.1000,"publicationDate":"2025-05-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Small RNAs as therapeutic agents: From catalytic motifs to regulatory pathways.\",\"authors\":\"John J Rossi, Saumya Das\",\"doi\":\"10.1016/j.ymthe.2025.03.053\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>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.</p>\",\"PeriodicalId\":19020,\"journal\":{\"name\":\"Molecular Therapy\",\"volume\":\" \",\"pages\":\"2238-2242\"},\"PeriodicalIF\":12.1000,\"publicationDate\":\"2025-05-07\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Molecular Therapy\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1016/j.ymthe.2025.03.053\",\"RegionNum\":1,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2025/4/2 0:00:00\",\"PubModel\":\"Epub\",\"JCR\":\"Q1\",\"JCRName\":\"BIOTECHNOLOGY & APPLIED MICROBIOLOGY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Molecular Therapy","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1016/j.ymthe.2025.03.053","RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2025/4/2 0:00:00","PubModel":"Epub","JCR":"Q1","JCRName":"BIOTECHNOLOGY & APPLIED MICROBIOLOGY","Score":null,"Total":0}
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.
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.