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http://bura.brunel.ac.uk/handle/2438/29221| Title: | Improving the assessment of risk factors relevant to potential carcinogenicity of gene therapies: a consensus paper |
| Authors: | Klapwijk, JC Del Rio Espinola, A Libertini, S Collin, P Fellows, MD Jobling, S Lynch, AM Martus, H Vickers, C Zeller, A Biasco, L Brugman, MH Bushmann, FD Cathomen, T Ertl, HCJ Gabriel, R Gao, G Jadlowsky, JK Kimber, I Lanz, TA Levine, BL Micklethwaite, KP Onodera, M Pizzurro, DM Reed, S Rothe, M Sabatino, DE Salk, JJ Schambach, A Themis, M Yuan, J |
| Keywords: | gene therapy vectors;carcinogenicity;genotoxicity;non-clinical safety |
| Issue Date: | 25-Jul-2024 |
| Publisher: | Mary Ann Liebert |
| Citation: | Klapwijk, J.C. et al. (2024) 'Improving the assessment of risk factors relevant to potential carcinogenicity of gene therapies: a consensus paper', Human Gene Therapy, 35 (15-16), pp. 527 - 542. doi: 10.1089/hum.2024.033. |
| 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 organised by NC3Rs/UKEMS (London, March 2023) to discuss this topic. This paper describes the consensus reached amongst 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 non-clinical toxicological assessment of GT products. |
| Description: | Supplementary Material is available online at: https://www.liebertpub.com/doi/full/10.1089/hum.2024.033#supplementary-materials . M.T. is a cofounder and Director of TestAVec Ltd., spun-out from Brunel University, and holds a patent concerning hInGetox “Method for Testing a Gene Therapy Vector” IP number PCT/GB2018/051937 with several collaborative projects in safety for gene therapy. |
| URI: | https://bura.brunel.ac.uk/handle/2438/29221 |
| DOI: | https://doi.org/10.1089/hum.2024.033 |
| ISSN: | 1043-0342 |
| Other Identifiers: | ORCiD: Jan C. Klapwijk https://orcid.org/0009-0007-6387-0192 ORCiD: Susan Jobling https://orcid.org/0000-0002-9322-9597 ORCiD: Catherine Vickers https://orcid.org/0009-0006-1642-5590 ORCiD: Toni Cathomen https://orcid.org/0000-0002-7757-4630 ORCiD: Hildegrund C.J. Ertl https://orcid.org/0000-0001-9795-5206 ORCiD: Axel Schambach https://orcid.org/0000-0003-2743-0070 ORCiD: Michael Themis https://orcid.org/0000-0002-6719-4271 |
| Appears in Collections: | Environment Dept of Life Sciences Research Papers |
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| File | Description | Size | Format | |
|---|---|---|---|---|
| FullText.pdf | Copyright © The Author(s) 2024. Published by Mary Ann Liebert, Inc. This Open Access article is distributed under the terms of the Creative Commons License [CC-BY] (https://creativecommons.org/licenses/by/4.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. | 677.78 kB | Adobe PDF | View/Open |
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