TGFβ Inhibitor A83-01 Enhances Murine HSPC Expansion for Gene Therapy

por Fleischauer, Jenni, Bastone, Antonella Lucia, Selich, Anton, John-Neek, Philipp, Weisskoeppel, Luisa, Schaudien, Dirk, Schambach, Axel, Rothe, Michael
Publicado 2023

Development of an in vitro genotoxicity assay to detect retroviral vector-induced lymphoid insertional mutants

por Bastone, Antonella L., Dziadek, Violetta, John-Neek, Philipp, Mansel, Friederike, Fleischauer, Jenni, Agyeman-Duah, Eric, Schaudien, Dirk, Dittrich-Breiholz, Oliver, Schwarzer, Adrian, Schambach, Axel, Rothe, Michael
Publicado 2023

Predicting genotoxicity of viral vectors for stem cell gene therapy using gene expression-based machine learning

por Schwarzer, Adrian, Talbot, Steven R., Selich, Anton, Morgan, Michael, Schott, Juliane W., Dittrich-Breiholz, Oliver, Bastone, Antonella L., Weigel, Bettina, Ha, Teng Cheong, Dziadek, Violetta, Gijsbers, Rik, Thrasher, Adrian J., Staal, Frank J.T., Gaspar, Hubert B., Modlich, Ute, Schambach, Axel, Rothe, Michael
Publicado 2021

Preclinical studies of efficacy thresholds and tolerability of a clinically ready lentiviral vector for pyruvate kinase deficiency treatment

por Navarro, Susana, Quintana-Bustamante, Oscar, Sanchez-Dominguez, Rebeca, Lopez-Manzaneda, Sergio, Ojeda-Perez, Isabel, Garcia-Torralba, Aida, Alberquilla, Omaira, Law, Kenneth, Beard, Brian C., Bastone, Antonella, Rothe, Michael, Villanueva, Mariela, Ramirez, Juan C., Fañanas-Baquero, Sara, Nieto-Romero, Virginia, Molinos-Vicente, Andrea, Gutierrez, Sonia, Nicoletti, Eileen, García-Bravo, María, Bueren, Juan A., Schwartz, Jonathan D., Segovia, Jose-Carlos
Publicado 2021