Improving the Assessment of Risk Factors Relevant to Potential Carcinogenicity of Gene Therapies: A Consensus Article.

IF 3.9 3区 医学 Q2 BIOTECHNOLOGY & APPLIED MICROBIOLOGY Human gene therapy Pub Date : 2024-08-01 DOI:10.1089/hum.2024.033
Jan C Klapwijk, Alberto Del Rio Espinola, Silvana Libertini, Philippe Collin, Mick D Fellows, Susan Jobling, Anthony M Lynch, HansJoerg Martus, Catherine Vickers, Andreas Zeller, Luca Biasco, Martijn H Brugman, Frederic D Bushmann, Toni Cathomen, Hildegrund C J Ertl, Richard Gabriel, Guangping Gao, Julie K Jadlowsky, Ian Kimber, Thomas A Lanz, Bruce L Levine, Kenneth P Micklethwaite, Masafumi Onodera, Daniella M Pizzurro, Simon Reed, Michael Rothe, Denise E Sabatino, Jesse J Salk, Axel Schambach, Michael Themis, Jing Yuan
{"title":"Improving the Assessment of Risk Factors Relevant to Potential Carcinogenicity of Gene Therapies: A Consensus Article.","authors":"Jan C Klapwijk, Alberto Del Rio Espinola, Silvana Libertini, Philippe Collin, Mick D Fellows, Susan Jobling, Anthony M Lynch, HansJoerg Martus, Catherine Vickers, Andreas Zeller, Luca Biasco, Martijn H Brugman, Frederic D Bushmann, Toni Cathomen, Hildegrund C J Ertl, Richard Gabriel, Guangping Gao, Julie K Jadlowsky, Ian Kimber, Thomas A Lanz, Bruce L Levine, Kenneth P Micklethwaite, Masafumi Onodera, Daniella M Pizzurro, Simon Reed, Michael Rothe, Denise E Sabatino, Jesse J Salk, Axel Schambach, Michael Themis, Jing Yuan","doi":"10.1089/hum.2024.033","DOIUrl":null,"url":null,"abstract":"<p><p>Regulators and industry are actively seeking improvements and alternatives to current models and approaches to evaluate potential carcinogenicity of gene therapies (GTs). A meeting of invited experts was organized by NC3Rs/UKEMS (London, March 2023) to discuss this topic. This article describes the consensus reached among delegates on the definition of vector genotoxicity, sources of uncertainty, suitable toxicological endpoints for genotoxic assessment of GTs, and future research needs. The collected recommendations should inform the further development of regulatory guidelines for the nonclinical toxicological assessment of GT products.</p>","PeriodicalId":13007,"journal":{"name":"Human gene therapy","volume":null,"pages":null},"PeriodicalIF":3.9000,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Human gene therapy","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1089/hum.2024.033","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"BIOTECHNOLOGY & APPLIED MICROBIOLOGY","Score":null,"Total":0}
引用次数: 0

Abstract

Regulators and industry are actively seeking improvements and alternatives to current models and approaches to evaluate potential carcinogenicity of gene therapies (GTs). A meeting of invited experts was organized by NC3Rs/UKEMS (London, March 2023) to discuss this topic. This article describes the consensus reached among delegates on the definition of vector genotoxicity, sources of uncertainty, suitable toxicological endpoints for genotoxic assessment of GTs, and future research needs. The collected recommendations should inform the further development of regulatory guidelines for the nonclinical toxicological assessment of GT products.

查看原文
分享 分享
微信好友 朋友圈 QQ好友 复制链接
本刊更多论文
改进与基因疗法潜在致癌性相关的风险因素评估:共识文件。
监管机构和业界正在积极寻求改进和替代现有模型和方法,以评估基因疗法 (GT) 的潜在致癌性。NC3Rs/UKEMS 组织了一次特邀专家会议(2023 年 3 月,伦敦)讨论这一主题。本文介绍了与会代表就载体基因毒性的定义、不确定性来源、GTs 基因毒性评估的合适毒理学终点以及未来研究需求达成的共识。收集到的建议将为进一步制定 GT 产品非临床毒理学评估监管指南提供参考。
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 去求助
来源期刊
Human gene therapy
Human gene therapy 医学-生物工程与应用微生物
CiteScore
6.50
自引率
4.80%
发文量
131
审稿时长
4-8 weeks
期刊介绍: Human Gene Therapy is the premier, multidisciplinary journal covering all aspects of gene therapy. The Journal publishes in-depth coverage of DNA, RNA, and cell therapies by delivering the latest breakthroughs in research and technologies. Human Gene Therapy provides a central forum for scientific and clinical information, including ethical, legal, regulatory, social, and commercial issues, which enables the advancement and progress of therapeutic procedures leading to improved patient outcomes, and ultimately, to curing diseases.
期刊最新文献
Neuroimaging Applications for the Delivery and Monitoring of Gene Therapy for Central Nervous System Diseases. Cyclosporin H Improves the Transduction of CD34+ Cells with an Anti-Sickling Globin Vector, a Possible Therapeutic Approach for Sickle Cell Disease. Adeno-Associated Virus Gene Transfer Ameliorates Progression of Skeletal Lesions in Mucopolysaccharidosis IVA Mice. Lentiviral Vector-Mediated Ex Vivo Hematopoietic Stem Cell Gene Therapy for Mucopolysaccharidosis IVA Murine Model. Suppression of CNS APOE4 Expression by miRNAs Delivered by the S2 AAVrh.10 Capsid-Modified AAV Vector.
×
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
现在去查看 取消
×
提示
确定
0
微信
客服QQ
Book学术公众号 扫码关注我们
反馈
×
意见反馈
请填写您的意见或建议
请填写您的手机或邮箱
已复制链接
已复制链接
快去分享给好友吧!
我知道了
×
扫码分享
扫码分享
Book学术官方微信
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术
文献互助 智能选刊 最新文献 互助须知 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 Book学术 All rights reserved.
ghs 京公网安备 11010802042870号 京ICP备2023020795号-1