Advances in Engineered Virus-Like Particles for Genome Editing and Therapy.

IF 8.5 2区 医学 Q1 IMMUNOLOGY
BioDrugs Pub Date : 2026-03-01 Epub Date: 2026-02-24 DOI:10.1007/s40259-026-00767-z
Se Hyeok Son, Seeone Woo, Ayeon Choi, Seungwon Ahn, Hee Chan Yoo
{"title":"Advances in Engineered Virus-Like Particles for Genome Editing and Therapy.","authors":"Se Hyeok Son, Seeone Woo, Ayeon Choi, Seungwon Ahn, Hee Chan Yoo","doi":"10.1007/s40259-026-00767-z","DOIUrl":null,"url":null,"abstract":"<p><p>Engineered virus-like particles (eVLPs) have emerged as a promising class of delivery systems for genome editing agents. By combining the efficient cellular entry of viral vectors with the safety advantages of nonviral platforms, eVLPs enable transient delivery of ribonucleoproteins such as Cas9, base editors, and prime editors. Successive design strategies, from rational engineering to directed optimization, have progressively improved particle assembly, cargo stability, and editing efficiency. In parallel, pseudotyping approaches have expanded the versatility of eVLPs for cell-type-specific targeting. Recent preclinical studies highlight their potential across diverse applications, ranging from monogenic disease models to complex disorders, and support their advancement toward clinical translation. This review summarizes the structural and production principles of eVLPs, surveys key advances in their development, and discusses therapeutic opportunities and future challenges for their application in genome editing.</p>","PeriodicalId":9022,"journal":{"name":"BioDrugs","volume":" ","pages":"317-333"},"PeriodicalIF":8.5000,"publicationDate":"2026-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13018093/pdf/","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"BioDrugs","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1007/s40259-026-00767-z","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2026/2/24 0:00:00","PubModel":"Epub","JCR":"Q1","JCRName":"IMMUNOLOGY","Score":null,"Total":0}
引用次数: 0

Abstract

Engineered virus-like particles (eVLPs) have emerged as a promising class of delivery systems for genome editing agents. By combining the efficient cellular entry of viral vectors with the safety advantages of nonviral platforms, eVLPs enable transient delivery of ribonucleoproteins such as Cas9, base editors, and prime editors. Successive design strategies, from rational engineering to directed optimization, have progressively improved particle assembly, cargo stability, and editing efficiency. In parallel, pseudotyping approaches have expanded the versatility of eVLPs for cell-type-specific targeting. Recent preclinical studies highlight their potential across diverse applications, ranging from monogenic disease models to complex disorders, and support their advancement toward clinical translation. This review summarizes the structural and production principles of eVLPs, surveys key advances in their development, and discusses therapeutic opportunities and future challenges for their application in genome editing.

Abstract Image

Abstract Image

Abstract Image

用于基因组编辑和治疗的工程病毒样颗粒研究进展。
工程病毒样颗粒(evlp)已成为一类有前途的基因组编辑剂递送系统。通过结合病毒载体的高效细胞进入和非病毒平台的安全优势,evlp能够瞬间递送核糖核蛋白,如Cas9、碱基编辑器和引物编辑器。连续的设计策略,从合理的工程到定向优化,逐步提高了粒子装配,货物稳定性和编辑效率。与此同时,假分型方法扩大了evlp用于细胞类型特异性靶向的多功能性。最近的临床前研究强调了它们在多种应用中的潜力,从单基因疾病模型到复杂疾病,并支持它们向临床转化的进展。本文综述了evlp的结构和产生原理,综述了其发展的关键进展,并讨论了其在基因组编辑中的应用的治疗机会和未来挑战。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
求助全文
约1分钟内获得全文 求助全文
来源期刊
BioDrugs
BioDrugs 医学-免疫学
CiteScore
12.60
自引率
2.90%
发文量
50
审稿时长
>12 weeks
期刊介绍: An essential resource for R&D professionals and clinicians with an interest in biologic therapies. BioDrugs covers the development and therapeutic application of biotechnology-based pharmaceuticals and diagnostic products for the treatment of human disease. BioDrugs offers a range of additional enhanced features designed to increase the visibility, readership and educational value of the journal’s content. Each article is accompanied by a Key Points summary, giving a time-efficient overview of the content to a wide readership. Articles may be accompanied by plain language summaries to assist patients, caregivers and others in understanding important medical advances. The journal also provides the option to include various other types of enhanced features including slide sets, videos and animations. All enhanced features are peer reviewed to the same high standard as the article itself. Peer review is conducted using Editorial Manager®, supported by a database of international experts. This database is shared with other Adis journals.
×
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
小红书