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Predicting genotoxicity of viral vectors for stem cell gene therapy using gene expression-based machine learning.
Schwarzer A, Talbot SR, Selich A, Morgan M, Schott JW, Dittrich-Breiholz O, Bastone AL, Weigel B, Ha TC, Dziadek V, Gijsbers R, Thrasher AJ, Staal FJT, Gaspar HB, Modlich U, Schambach A, Rothe M. Schwarzer A, et al. Among authors: bastone al. Mol Ther. 2021 Dec 1;29(12):3383-3397. doi: 10.1016/j.ymthe.2021.06.017. Epub 2021 Jun 24. Mol Ther. 2021. PMID: 34174440 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: bastone al. Cells. 2023 Jul 31;12(15):1978. doi: 10.3390/cells12151978. Cells. 2023. PMID: 37566057 Free PMC article.
Preclinical studies of efficacy thresholds and tolerability of a clinically ready lentiviral vector for pyruvate kinase deficiency treatment.
Navarro S, Quintana-Bustamante O, Sanchez-Dominguez R, Lopez-Manzaneda S, Ojeda-Perez I, Garcia-Torralba A, Alberquilla O, Law K, Beard BC, Bastone A, Rothe M, Villanueva M, Ramirez JC, Fañanas-Baquero S, Nieto-Romero V, Molinos-Vicente A, Gutierrez S, Nicoletti E, García-Bravo M, Bueren JA, Schwartz JD, Segovia JC. Navarro S, et al. Mol Ther Methods Clin Dev. 2021 Jul 29;22:350-359. doi: 10.1016/j.omtm.2021.07.006. eCollection 2021 Sep 10. Mol Ther Methods Clin Dev. 2021. PMID: 34514027 Free PMC article.