rna疗法:临床研究的前沿进展

IF 2.6 4区 医学 Q2 PHARMACOLOGY & PHARMACY
Débora Ferreira, Ligia R. Rodrigues
{"title":"rna疗法:临床研究的前沿进展","authors":"Débora Ferreira,&nbsp;Ligia R. Rodrigues","doi":"10.1002/adtp.202500082","DOIUrl":null,"url":null,"abstract":"<p>Over the years, the relevance of gene therapy has increased as an alternative to conventional drugs. Pharmaceutical industries have been directing their efforts toward the creation of novel gene-based therapies for a broad range of diseases. RNA-based therapeutics can efficiently trigger gene silencing and, despite the initial research setbacks, this technology laid the groundwork for advancements in RNA-based drug design that ultimately contributed to the remarkably rapid advance of messenger RNA (mRNA) vaccines to combat the COVID-19 pandemic. In light of this information, RNA medicines are currently well-positioned to have a noteworthy impact in clinical practice. Various RNA medicines have received clinical approval, while others are still in the research phase or undergoing preclinical trials. In this review, an overview of RNA-based therapeutics is aimed to provide, including antisense oligonucleotides and RNA interference-based mechanisms, mRNA-encoded approaches, aptamers, and clustered regularly interspaced short palindromic repeats (CRISPR-Cas)-mediated genome editing. Furthermore, the commonly used chemistries in drug delivery systems and strategies for targeting specific cells, as well as the key nanocarriers used in RNA loading, are explored. The main advances and current challenges will be thoroughly summarized.</p>","PeriodicalId":7284,"journal":{"name":"Advanced Therapeutics","volume":"8 8","pages":""},"PeriodicalIF":2.6000,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"RNA-Based Therapeutics: Cutting-Edge Advances in Clinical Research\",\"authors\":\"Débora Ferreira,&nbsp;Ligia R. Rodrigues\",\"doi\":\"10.1002/adtp.202500082\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p>Over the years, the relevance of gene therapy has increased as an alternative to conventional drugs. Pharmaceutical industries have been directing their efforts toward the creation of novel gene-based therapies for a broad range of diseases. RNA-based therapeutics can efficiently trigger gene silencing and, despite the initial research setbacks, this technology laid the groundwork for advancements in RNA-based drug design that ultimately contributed to the remarkably rapid advance of messenger RNA (mRNA) vaccines to combat the COVID-19 pandemic. In light of this information, RNA medicines are currently well-positioned to have a noteworthy impact in clinical practice. Various RNA medicines have received clinical approval, while others are still in the research phase or undergoing preclinical trials. In this review, an overview of RNA-based therapeutics is aimed to provide, including antisense oligonucleotides and RNA interference-based mechanisms, mRNA-encoded approaches, aptamers, and clustered regularly interspaced short palindromic repeats (CRISPR-Cas)-mediated genome editing. Furthermore, the commonly used chemistries in drug delivery systems and strategies for targeting specific cells, as well as the key nanocarriers used in RNA loading, are explored. The main advances and current challenges will be thoroughly summarized.</p>\",\"PeriodicalId\":7284,\"journal\":{\"name\":\"Advanced Therapeutics\",\"volume\":\"8 8\",\"pages\":\"\"},\"PeriodicalIF\":2.6000,\"publicationDate\":\"2025-07-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Advanced Therapeutics\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://advanced.onlinelibrary.wiley.com/doi/10.1002/adtp.202500082\",\"RegionNum\":4,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q2\",\"JCRName\":\"PHARMACOLOGY & PHARMACY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Advanced Therapeutics","FirstCategoryId":"3","ListUrlMain":"https://advanced.onlinelibrary.wiley.com/doi/10.1002/adtp.202500082","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"PHARMACOLOGY & PHARMACY","Score":null,"Total":0}
引用次数: 0

摘要

多年来,基因治疗作为一种传统药物的替代品,其相关性有所增加。制药行业一直致力于开发针对多种疾病的新型基因疗法。基于RNA的治疗方法可以有效地触发基因沉默,尽管最初的研究受挫,但这项技术为基于RNA的药物设计的进步奠定了基础,最终促进了信使RNA (mRNA)疫苗的快速发展,以对抗COVID-19大流行。根据这些信息,RNA药物目前在临床实践中具有显著的影响。各种RNA药物已经获得临床批准,而其他药物仍处于研究阶段或正在进行临床前试验。在这篇综述中,综述了基于RNA的治疗方法,包括反义寡核苷酸和基于RNA干扰的机制、mrna编码方法、适体和聚集规律间隔短回文重复序列(CRISPR-Cas)介导的基因组编辑。此外,还探讨了药物递送系统中常用的化学物质和靶向特定细胞的策略,以及RNA装载中使用的关键纳米载体。本文将对目前的主要进展和挑战进行全面总结。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

RNA-Based Therapeutics: Cutting-Edge Advances in Clinical Research

RNA-Based Therapeutics: Cutting-Edge Advances in Clinical Research

RNA-Based Therapeutics: Cutting-Edge Advances in Clinical Research

RNA-Based Therapeutics: Cutting-Edge Advances in Clinical Research

RNA-Based Therapeutics: Cutting-Edge Advances in Clinical Research

Over the years, the relevance of gene therapy has increased as an alternative to conventional drugs. Pharmaceutical industries have been directing their efforts toward the creation of novel gene-based therapies for a broad range of diseases. RNA-based therapeutics can efficiently trigger gene silencing and, despite the initial research setbacks, this technology laid the groundwork for advancements in RNA-based drug design that ultimately contributed to the remarkably rapid advance of messenger RNA (mRNA) vaccines to combat the COVID-19 pandemic. In light of this information, RNA medicines are currently well-positioned to have a noteworthy impact in clinical practice. Various RNA medicines have received clinical approval, while others are still in the research phase or undergoing preclinical trials. In this review, an overview of RNA-based therapeutics is aimed to provide, including antisense oligonucleotides and RNA interference-based mechanisms, mRNA-encoded approaches, aptamers, and clustered regularly interspaced short palindromic repeats (CRISPR-Cas)-mediated genome editing. Furthermore, the commonly used chemistries in drug delivery systems and strategies for targeting specific cells, as well as the key nanocarriers used in RNA loading, are explored. The main advances and current challenges will be thoroughly summarized.

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
Advanced Therapeutics
Advanced Therapeutics Pharmacology, Toxicology and Pharmaceutics-Pharmaceutical Science
CiteScore
7.10
自引率
2.20%
发文量
130
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
确定
请完成安全验证×
copy
已复制链接
快去分享给好友吧!
我知道了
右上角分享
点击右上角分享
0
联系我们:info@booksci.cn Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。 Copyright © 2023 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号:604180095
Book学术官方微信