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":"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。","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":"{\"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}","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}
Improving the Assessment of Risk Factors Relevant to Potential Carcinogenicity of Gene Therapies: A Consensus Article.
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.
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.
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