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Page 1
Development of an in vitro genotoxicity assay to detect retroviral vector-induced lymphoid insertional mutants.
Bastone AL, Dziadek V, John-Neek P, Mansel F, Fleischauer J, Agyeman-Duah E, Schaudien D, Dittrich-Breiholz O, Schwarzer A, Schambach A, Rothe M. Bastone AL, et al. Among authors: rothe m. Mol Ther Methods Clin Dev. 2023 Aug 22;30:515-533. doi: 10.1016/j.omtm.2023.08.017. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37693949 Free PMC article.
Persistent hematopoietic polyclonality after lentivirus-mediated gene therapy for Fabry disease.
Saleh AH, Rothe M, Barber DL, McKillop WM, Fraser G, Morel CF, Schambach A, Auray-Blais C, West ML, Khan A, Fowler DH, Rupar CA, Foley R, Medin JA, Keating A. Saleh AH, et al. Among authors: rothe m. Mol Ther Methods Clin Dev. 2023 Jan 18;28:262-271. doi: 10.1016/j.omtm.2023.01.003. eCollection 2023 Mar 9. Mol Ther Methods Clin Dev. 2023. PMID: 36816757 Free PMC article.
TGFβ Inhibitor A83-01 Enhances Murine HSPC Expansion for Gene Therapy.
Fleischauer J, Bastone AL, Selich A, John-Neek P, Weisskoeppel L, Schaudien D, Schambach A, Rothe M. Fleischauer J, et al. Among authors: rothe m. Cells. 2023 Jul 31;12(15):1978. doi: 10.3390/cells12151978. Cells. 2023. PMID: 37566057 Free PMC article.
Pharmacological and pre-clinical safety profile of rSIV.F/HN, a hybrid lentiviral vector for cystic fibrosis gene therapy.
Moiseenko A, Sinadinos A, Sergijenko A, Pineault K, Saleh A, Nekola K, Strang N, Eleftheraki A, Boyd AC, Davies JC, Gill DR, Hyde SC, McLachlan G, Rath T, Rothe M, Schambach A, Hobbie S, Schuler M, Maier U, Thomas MJ, Mennerich D, Schmidt M, Griesenbach U, Alton EWFW, Kreuz S. Moiseenko A, et al. Among authors: rothe m. Eur Respir J. 2024 Nov 21:2301683. doi: 10.1183/13993003.01683-2023. Online ahead of print. Eur Respir J. 2024. PMID: 39174284 Free article.
Preclinical lentiviral hematopoietic stem cell gene therapy corrects Pompe disease-related muscle and neurological manifestations.
Yoon JK, Schindler JW, Loperfido M, Baricordi C, DeAndrade MP, Jacobs ME, Treleaven C, Plasschaert RN, Yan A, Barese CN, Dogan Y, Chen VP, Fiorini C, Hull F, Barbarossa L, Unnisa Z, Ivanov D, Kutner RH, Guda S, Oborski C, Maiwald T, Michaud V, Rothe M, Schambach A, Pfeifer R, Mason C, Biasco L, van Til NP. Yoon JK, et al. Among authors: rothe m. Mol Ther. 2024 Nov 6;32(11):3847-3864. doi: 10.1016/j.ymthe.2024.09.024. Epub 2024 Sep 17. Mol Ther. 2024. PMID: 39295144
Haematopoietic gene therapy of non-conditioned patients with Fanconi anaemia-A: results from open-label phase 1/2 (FANCOLEN-1) and long-term clinical trials.
Río P, Zubicaray J, Navarro S, Gálvez E, Sánchez-Domínguez R, Nicoletti E, Sebastián E, Rothe M, Pujol R, Bogliolo M, John-Neek P, Bastone AL, Schambach A, Wang W, Schmidt M, Larcher L, Segovia JC, Yáñez RM, Alberquilla O, Díez B, Fernández-García M, García-García L, Ramírez M, Galy A, Lefrere F, Cavazzana M, Leblanc T, García de Andoin N, López-Almaraz R, Catalá A, Barquinero J, Rodríguez-Perales S, Rao G, Surrallés J, Soulier J, Díaz-de-Heredia C, Schwartz JD, Sevilla J, Bueren JA; FANCOLEN-1 gene therapy investigators. Río P, et al. Among authors: rothe m. Lancet. 2025 Dec 21;404(10471):2584-2592. doi: 10.1016/S0140-6736(24)01880-4. Epub 2024 Dec 3. Lancet. 2025. PMID: 39642902 Clinical Trial.
Preclinical toxicity analyses of lentiviral vectors expressing the HIV-1 LTR-specific designer-recombinase Brec1.
Beschorner N, Künzle P, Voges M, Hauber I, Indenbirken D, Nakel J, Virdi S, Bradtke P, Lory NC, Rothe M, Paszkowski-Rogacz M, Buchholz F, Grundhoff A, Schambach A, Thirion C, Mittrücker HW, Schulze Zur Wiesch J, Hauber J, Chemnitz J. Beschorner N, et al. Among authors: rothe m. PLoS One. 2024 Mar 8;19(3):e0298542. doi: 10.1371/journal.pone.0298542. eCollection 2024. PLoS One. 2024. PMID: 38457474 Free PMC article.
Improving the Assessment of Risk Factors Relevant to Potential Carcinogenicity of Gene Therapies: A Consensus Article.
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. Klapwijk JC, et al. Among authors: rothe m. Hum Gene Ther. 2024 Aug;35(15-16):527-542. doi: 10.1089/hum.2024.033. Hum Gene Ther. 2024. PMID: 39049734 Review.
Palmitoylcarnitine impairs immunity in decompensated cirrhosis.
Zhang IW, Sánchez-Rodríguez MB, López-Vicario C, Casulleras M, Duran-Güell M, Flores-Costa R, Aguilar F, Rothe M, Segalés P, García-Ruiz C, Fernández-Checa JC, Trebicka J, Arroyo V, Clària J. Zhang IW, et al. Among authors: rothe m. JHEP Rep. 2024 Aug 22;6(11):101187. doi: 10.1016/j.jhepr.2024.101187. eCollection 2024 Nov. JHEP Rep. 2024. PMID: 39524205 Free PMC article.
442 results