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Page 1
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: schambach a. 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.
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: schambach a. Hum Gene Ther. 2024 Aug;35(15-16):527-542. doi: 10.1089/hum.2024.033. Hum Gene Ther. 2024. PMID: 39049734 Review.
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: schambach a. 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.
Correction for Gupta et al., "Bromo- and Extraterminal Domain Chromatin Regulators Serve as Cofactors for Murine Leukemia Virus Integration".
Gupta SS, Maetzig T, Maertens GN, Sharif A, Rothe M, Weidner-Glunde M, Galla M, Schambach A, Cherepanov P, Schulz TF. Gupta SS, et al. Among authors: schambach a. J Virol. 2024 Apr 16;98(4):e0013524. doi: 10.1128/jvi.00135-24. Epub 2024 Mar 13. J Virol. 2024. PMID: 38477531 Free PMC article. No abstract available.
Anti-Mesothelin CAR-NK cells as a novel targeted therapy against cervical cancer.
Kutle I, Polten R, Stalp JL, Hachenberg J, Todzey F, Hass R, Zimmermann K, von der Ohe J, von Kaisenberg C, Neubert L, Kamp JC, Schaudien D, Seyda AK, Hillemanns P, Klapdor R, Morgan MA, Schambach A. Kutle I, et al. Among authors: schambach a. Front Immunol. 2024 Dec 16;15:1485461. doi: 10.3389/fimmu.2024.1485461. eCollection 2024. Front Immunol. 2024. PMID: 39781381 Free PMC article.
Blood-generating heart-forming organoids recapitulate co-development of the human haematopoietic system and the embryonic heart.
Dardano M, Kleemiß F, Kosanke M, Lang D, Wilson L, Franke A, Teske J, Shivaraj A, de la Roche J, Fischer M, Lange L, Schambach A, Drakhlis L, Zweigerdt R. Dardano M, et al. Among authors: schambach a. Nat Cell Biol. 2024 Nov;26(11):1984-1996. doi: 10.1038/s41556-024-01526-4. Epub 2024 Oct 8. Nat Cell Biol. 2024. PMID: 39379702 Free PMC 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: schambach a. 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
369 results