长非编码 RNA 的靶向和工程设计用于癌症治疗。

IF 39.1 1区生物学 Q1 GENETICS & HEREDITY

Nature Reviews Genetics Pub Date : 2024-02-29 DOI:10.1038/s41576-024-00693-2

Michela Coan, Simon Haefliger, Samir Ounzain, Rory Johnson

{"title":"长非编码 RNA 的靶向和工程设计用于癌症治疗。","authors":"Michela Coan, Simon Haefliger, Samir Ounzain, Rory Johnson","doi":"10.1038/s41576-024-00693-2","DOIUrl":null,"url":null,"abstract":"RNA therapeutics (RNATx) aim to treat diseases, including cancer, by targeting or employing RNA molecules for therapeutic purposes. Amongst the most promising targets are long non-coding RNAs (lncRNAs), which regulate oncogenic molecular networks in a cell type-restricted manner. lncRNAs are distinct from protein-coding genes in important ways that increase their therapeutic potential yet also present hurdles to conventional clinical development. Advances in genome editing, oligonucleotide chemistry, multi-omics and RNA engineering are paving the way for efficient and cost-effective lncRNA-focused drug discovery pipelines. In this Review, we present the emerging field of lncRNA therapeutics for oncology, with emphasis on the unique strengths and challenges of lncRNAs within the broader RNATx framework. We outline the necessary steps for lncRNA therapeutics to deliver effective, durable, tolerable and personalized treatments for cancer. Therapeutics that target long non-coding RNAs (lncRNAs) are promising treatments for cancer. In this Review, the authors discuss how technological advances have helped improve drug discovery pipelines for lncRNAs and overview their strengths and challenges as oncological therapeutics.","PeriodicalId":19067,"journal":{"name":"Nature Reviews Genetics","volume":"25 8","pages":"578-595"},"PeriodicalIF":39.1000,"publicationDate":"2024-02-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Targeting and engineering long non-coding RNAs for cancer therapy\",\"authors\":\"Michela Coan, Simon Haefliger, Samir Ounzain, Rory Johnson\",\"doi\":\"10.1038/s41576-024-00693-2\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"RNA therapeutics (RNATx) aim to treat diseases, including cancer, by targeting or employing RNA molecules for therapeutic purposes. Amongst the most promising targets are long non-coding RNAs (lncRNAs), which regulate oncogenic molecular networks in a cell type-restricted manner. lncRNAs are distinct from protein-coding genes in important ways that increase their therapeutic potential yet also present hurdles to conventional clinical development. Advances in genome editing, oligonucleotide chemistry, multi-omics and RNA engineering are paving the way for efficient and cost-effective lncRNA-focused drug discovery pipelines. In this Review, we present the emerging field of lncRNA therapeutics for oncology, with emphasis on the unique strengths and challenges of lncRNAs within the broader RNATx framework. We outline the necessary steps for lncRNA therapeutics to deliver effective, durable, tolerable and personalized treatments for cancer. Therapeutics that target long non-coding RNAs (lncRNAs) are promising treatments for cancer. In this Review, the authors discuss how technological advances have helped improve drug discovery pipelines for lncRNAs and overview their strengths and challenges as oncological therapeutics.\",\"PeriodicalId\":19067,\"journal\":{\"name\":\"Nature Reviews Genetics\",\"volume\":\"25 8\",\"pages\":\"578-595\"},\"PeriodicalIF\":39.1000,\"publicationDate\":\"2024-02-29\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Nature Reviews Genetics\",\"FirstCategoryId\":\"99\",\"ListUrlMain\":\"https://www.nature.com/articles/s41576-024-00693-2\",\"RegionNum\":1,\"RegionCategory\":\"生物学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"GENETICS & HEREDITY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Nature Reviews Genetics","FirstCategoryId":"99","ListUrlMain":"https://www.nature.com/articles/s41576-024-00693-2","RegionNum":1,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"GENETICS & HEREDITY","Score":null,"Total":0}

引用次数: 0

摘要

RNA 疗法（RNATx）旨在通过靶向或利用 RNA 分子达到治疗目的来治疗疾病，包括癌症。长非编码 RNA（lncRNA）是最有希望的靶点之一，它们以细胞类型受限的方式调控致癌分子网络。lncRNA 在一些重要方面有别于蛋白编码基因，这增加了它们的治疗潜力，但也给传统的临床开发带来了障碍。基因组编辑、寡核苷酸化学、多组学和 RNA 工程等领域的进步正在为高效、低成本的 lncRNA 药物研发铺平道路。在这篇综述中，我们将介绍用于肿瘤学的 lncRNA 疗法这一新兴领域，重点是 lncRNA 在更广泛的 RNATx 框架内的独特优势和挑战。我们概述了 lncRNA 疗法为癌症提供有效、持久、可耐受和个性化治疗的必要步骤。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

Targeting and engineering long non-coding RNAs for cancer therapy

查看原文本刊更多论文

Targeting and engineering long non-coding RNAs for cancer therapy

RNA therapeutics (RNATx) aim to treat diseases, including cancer, by targeting or employing RNA molecules for therapeutic purposes. Amongst the most promising targets are long non-coding RNAs (lncRNAs), which regulate oncogenic molecular networks in a cell type-restricted manner. lncRNAs are distinct from protein-coding genes in important ways that increase their therapeutic potential yet also present hurdles to conventional clinical development. Advances in genome editing, oligonucleotide chemistry, multi-omics and RNA engineering are paving the way for efficient and cost-effective lncRNA-focused drug discovery pipelines. In this Review, we present the emerging field of lncRNA therapeutics for oncology, with emphasis on the unique strengths and challenges of lncRNAs within the broader RNATx framework. We outline the necessary steps for lncRNA therapeutics to deliver effective, durable, tolerable and personalized treatments for cancer. Therapeutics that target long non-coding RNAs (lncRNAs) are promising treatments for cancer. In this Review, the authors discuss how technological advances have helped improve drug discovery pipelines for lncRNAs and overview their strengths and challenges as oncological therapeutics.

求助全文

通过发布文献求助，成功后即可免费获取论文全文。去求助

来源期刊

Nature Reviews Genetics 生物-遗传学

CiteScore

57.40

自引率

0.50%

发文量

113

审稿时长

6-12 weeks

期刊介绍： At Nature Reviews Genetics, our goal is to be the leading source of reviews and commentaries for the scientific communities we serve. We are dedicated to publishing authoritative articles that are easily accessible to our readers. We believe in enhancing our articles with clear and understandable figures, tables, and other display items. Our aim is to provide an unparalleled service to authors, referees, and readers, and we are committed to maximizing the usefulness and impact of each article we publish. Within our journal, we publish a range of content including Research Highlights, Comments, Reviews, and Perspectives that are relevant to geneticists and genomicists. With our broad scope, we ensure that the articles we publish reach the widest possible audience. As part of the Nature Reviews portfolio of journals, we strive to uphold the high standards and reputation associated with this esteemed collection of publications.