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

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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.

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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。

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来源期刊

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.