Gene therapy for Wiskott-Aldrich Syndrome—Long-term reconstitution and clinical benefits, but increased risk for leukemogenesis

di Braun, Christian Joerg, Witzel, Maximilian, Paruzynski, Anna, Boztug, Kaan, von Kalle, Christof, Schmidt, Manfred, Klein, Christoph
Pubblicato in Rare Dis (2014)

Cell Cycle Status of CD34(+) Hemopoietic Stem Cells Determines Lentiviral Integration in Actively Transcribed and Development-related Genes

di Papanikolaou, Eleni, Paruzynski, Anna, Kasampalidis, Ioannis, Deichmann, Annette, Stamateris, Evangelos, Schmidt, Manfred, von Kalle, Christof, Anagnou, Nicholas P
Pubblicato in Mol Ther (2015)

Bioinformatic Clonality Analysis of Next-Generation Sequencing-Derived Viral Vector Integration Sites

di Arens, Anne, Appelt, Jens-Uwe, Bartholomae, Cynthia C., Gabriel, Richard, Paruzynski, Anna, Gustafson, Derek, Cartier, Nathalie, Aubourg, Patrick, Deichmann, Annette, Glimm, Hanno, von Kalle, Christof, Schmidt, Manfred
Pubblicazione 2012

High-throughput monitoring of integration site clonality in preclinical and clinical gene therapy studies

di Giordano, Frank A, Appelt, Jens-Uwe, Link, Barbara, Gerdes, Sebastian, Lehrer, Christina, Scholz, Simone, Paruzynski, Anna, Roeder, Ingo, Wenz, Frederik, Glimm, Hanno, von Kalle, Christof, Grez, Manuel, Schmidt, Manfred, Laufs, Stephanie
Pubblicato in Mol Ther Methods Clin Dev (2015)

High-resolution analysis of the human T-cell receptor repertoire

di Ruggiero, Eliana, Nicolay, Jan P., Fronza, Raffaele, Arens, Anne, Paruzynski, Anna, Nowrouzi, Ali, Ürenden, Gökçe, Lulay, Christina, Schneider, Sven, Goerdt, Sergij, Glimm, Hanno, Krammer, Peter H., Schmidt, Manfred, von Kalle, Christof
Pubblicato in Nat Commun (2015)

Retroviral Gene Therapy for X-linked Chronic Granulomatous Disease: Results From Phase I/II Trial

di Kang, Hyoung Jin, Bartholomae, Cynthia C, Paruzynski, Anna, Arens, Anne, Kim, Sujeong, Yu, Seung Shin, Hong, Youngtae, Joo, Chang-Wan, Yoon, Nam-Kyung, Rhim, Jung-Woo, Kim, Joong Gon, Von Kalle, Christof, Schmidt, Manfred, Kim, Sunyoung, Ahn, Hyo Seop
Pubblicazione 2011

Lentiviral Vector Integration Profiles Differ in Rodent Postmitotic Tissues

di Bartholomae, Cynthia C, Arens, Anne, Balaggan, Kamaljit S, Yáñez-Muñoz, Rafael J, Montini, Eugenio, Howe, Steven J, Paruzynski, Anna, Korn, Bernhard, Appelt, Jens Uwe, MacNeil, Angus, Cesana, Daniela, Abel, Ulrich, Glimm, Hanno, Naldini, Luigi, Ali, Robin R, Thrasher, Adrian J, von Kalle, Christof, Schmidt, Manfred
Pubblicazione 2011

The Fetal Mouse Is a Sensitive Genotoxicity Model That Exposes Lentiviral-associated Mutagenesis Resulting in Liver Oncogenesis

di Nowrouzi, Ali, Cheung, Wing T, Li, Tingting, Zhang, Xuegong, Arens, Anne, Paruzynski, Anna, Waddington, Simon N, Osejindu, Emma, Reja, Safia, von Kalle, Christof, Wang, Yoahe, Al-Allaf, Faisal, Gregory, Lisa, Themis, Matthew, Holder, Maxine, Dighe, Niraja, Ruthe, Alaine, Buckley, Suzanne MK, Bigger, Brian, Montini, Eugenio, Thrasher, Adrian J, Andrews, Robert, Roberts, Terry P, Newbold, Robert F, Coutelle, Charles, Schmidt, Manfred, Themis, Mike
Pubblicazione 2013

Insertion Sites in Engrafted Cells Cluster Within a Limited Repertoire of Genomic Areas After Gammaretroviral Vector Gene Therapy

di Deichmann, Annette, Brugman, Martijn H, Bartholomae, Cynthia C, Schwarzwaelder, Kerstin, Verstegen, Monique MA, Howe, Steven J, Arens, Anne, Ott, Marion G, Hoelzer, Dieter, Seger, Reinhard, Grez, Manuel, Hacein-Bey-Abina, Salima, Cavazzana-Calvo, Marina, Fischer, Alain, Paruzynski, Anna, Gabriel, Richard, Glimm, Hanno, Abel, Ulrich, Cattoglio, Claudia, Mavilio, Fulvio, Cassani, Barbara, Aiuti, Alessandro, Dunbar, Cynthia E, Baum, Christopher, Gaspar, H Bobby, Thrasher, Adrian J, von Kalle, Christof, Schmidt, Manfred, Wagemaker, Gerard
Pubblicazione 2011