Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1 , PRDM16 or SETBP1
von Kalle, Christof, Grez, Manuel, Ott, Marion G, Schmidt, Manfred, Schwarzwaelder, Kerstin, Stein, Stefan, Siler, Ulrich, Koehl, Ulrike, Glimm, Hanno, Kühlcke, Klaus, Schilz, Andrea, Kunkel, Hana, Naundorf, Sonja, Brinkmann, Andrea, Deichmann, Annette, Fischer, Marlene, Ball, Claudia, Pilz, Ingo, Dunbar, Cynthia, Du, Yang, Jenkins, Nancy A, Copeland, Neal G, Lüthi, Ursula, Hassan, Moustapha, Thrasher, Adrian J, Hoelzer, Dieter, Seger, Reinhard
Published in Nature medicine (01.04.2006)
Published in Nature medicine (01.04.2006)
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Journal Article
Venetoclax and idasanutlin in relapsed/refractory AML: a nonrandomized, open-label phase 1b trial
Daver, Naval G., Dail, Monique, Garcia, Jacqueline S., Jonas, Brian A., Yee, Karen W. L., Kelly, Kevin R., Vey, Norbert, Assouline, Sarit, Roboz, Gail J., Paolini, Stefania, Pollyea, Daniel A., Tafuri, Agostino, Brandwein, Joseph M., Pigneux, Arnaud, Powell, Bayard L., Fenaux, Pierre, Olin, Rebecca L., Visani, Giuseppe, Martinelli, Giovanni, Onishi, Maika, Wang, Jue, Huang, Weize, Green, Cherie, Ott, Marion G., Hong, Wan-Jen, Konopleva, Marina Y., Andreeff, Michael
Published in Blood (16.03.2023)
Published in Blood (16.03.2023)
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Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease
Karakaya, Kadin, Ball, Claudia, Ott, Marion G, Seger, Reinhard, Schultze-Strasser, Stephan, Grez, Manuel, Koehl, Ulrike, Preiss, Carolin, Kinner, Andrea, von Kalle, Christof, Stein, Stefan, Schwäble, Joachim, Kühlcke, Klaus, Göhring, Gudrun, Jauch, Anna, Martin, Hans, Schwarzwaelder, Kerstin, Schmidt, Manfred, Yang, Rongxi, Thrasher, Adrian J, Tchatchou, Sandrine, Glimm, Hanno, Krämer, Alwin, Schlegelberger, Brigitte, Reinecke, Petra, Hofmann, Wolf-Karsten, Burwinkel, Barbara, Hoelzer, Dieter
Published in Nature medicine (01.02.2010)
Published in Nature medicine (01.02.2010)
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Journal Article
Humoral response to catumaxomab correlates with clinical outcome: Results of the pivotal phase II/III study in patients with malignant ascites
Ott, Marion G., Marmé, Frederik, Moldenhauer, Gerhard, Lindhofer, Horst, Hennig, Michael, Spannagl, Rolf, Essing, Mirko M., Linke, Rolf, Seimetz, Diane
Published in International journal of cancer (01.05.2012)
Published in International journal of cancer (01.05.2012)
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Journal Article
Venetoclax and Cobimetinib in Relapsed/Refractory AML: A Phase 1b Trial
Konopleva, Marina Y., Dail, Monique, Daver, Naval G., Garcia, Jacqueline S., Jonas, Brian A., Yee, Karen W.L., Kelly, Kevin R., Vey, Norbert, Assouline, Sarit, Roboz, Gail J., Paolini, Stefania, Pollyea, Daniel A., Tafuri, Agostino, Brandwein, Joseph M., Pigneux, Arnaud, Powell, Bayard L., Fenaux, Pierre, Olin, Rebecca L., Visani, Giuseppe, Martinelli, Giovanni, Onishi, Maika, Wang, Jue, Huang, Weize, Dunshee, Diana R., Hamidi, Habib, Ott, Marion G., Hong, Wan-Jen, Andreeff, Michael
Published in Clinical lymphoma, myeloma and leukemia (01.06.2024)
Published in Clinical lymphoma, myeloma and leukemia (01.06.2024)
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Journal Article
Insertion Sites in Engrafted Cells Cluster Within a Limited Repertoire of Genomic Areas After Gammaretroviral Vector Gene Therapy
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
Published in Molecular therapy (01.11.2011)
Published in Molecular therapy (01.11.2011)
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Journal Article
Advances in the treatment of Chronic Granulomatous Disease by gene therapy
Ott, Marion G, Seger, Reinhard, Stein, Stefan, Siler, Ulrich, Hoelzer, Dieter, Grez, Manuel
Published in Current gene therapy (01.06.2007)
Published in Current gene therapy (01.06.2007)
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Journal Article
723. In Vivo Expansion of MDS1/EVI1, PRDM16 and SETBP1 Integration Clones in Successful Chronic Granulomatous Disease (CGD) Gene Therapy Trial
Schwarzwaelder, Kerstin, Schmidt, Manfred, Ott, Marion G., Stein, Stefan, Glimm, Hanno, Deichmann, Annette, Siler, Ulrich, Hoelzer, Dieter, Seger, Reinhard, Grez, Manuel, von Kalle, Christof
Published in Molecular therapy (01.05.2006)
Published in Molecular therapy (01.05.2006)
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Journal Article
Updated Results from the Venetoclax (Ven) in Combination with Idasanutlin (Idasa) Arm of a Phase 1b Trial in Elderly Patients (Pts) with Relapsed or Refractory (R/R) AML Ineligible for Cytotoxic Chemotherapy
Daver, Naval G., Garcia, Jacqueline S, Jonas, Brian A, Kelly, Kevin R, Assouline, Sarit, Brandwein, Joseph M, Fenaux, Pierre, Olin, Rebecca L., Martinelli, Giovanni, Paolini, Stefania, Pigneux, Arnaud, Pollyea, Daniel A, Powell, Bayard L, Roboz, Gail J., Tafuri, Agostino, Vey, Norbert, Visani, Giuseppe, Yee, Karen W.L., Dail, Monique, Green, Cherie, Kirschbrown, Whitney P, Hong, Wan-Jen, Ott, Marion G, Onishi, Maika, Wang, Jue, Konopleva, Marina Y, Andreeff, Michael
Published in Blood (13.11.2019)
Published in Blood (13.11.2019)
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Safety, Efficacy, Pharmacokinetic (PK) and Biomarker Analyses of BCL2 Inhibitor Venetoclax (Ven) Plus MDM2 Inhibitor Idasanutlin (idasa) in Patients (pts) with Relapsed or Refractory (R/R) AML: A Phase Ib, Non-Randomized, Open-Label Study
Daver, Naval G., Pollyea, Daniel A, Garcia, Jacqueline S., Jonas, Brian A., Yee, Karen W.L., Fenaux, Pierre, Assouline, Sarit, Vey, Norbert, Olin, Rebecca, Roboz, Gail J., Tafuri, Agostino, Visani, Giuseppe, Kirschbrown, Whitney, Green, Cherie, Ma, Connie, Dail, Monique, Wang, Jue, Ott, Marion G, Mobasher, Mehrdad, Phuong, Patrick, Hong, Wan-Jen, Konopleva, Marina Y., Andreeff, Michael
Published in Blood (29.11.2018)
Published in Blood (29.11.2018)
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Journal Article
Gene therapy for chronic granulomatous disease
Stein, Stefan, Siler, Ulrich, Ott, Marion G, Seger, Reinhard, Grez, Maunel
Published in Current opinion in molecular therapeutics (01.10.2006)
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Published in Current opinion in molecular therapeutics (01.10.2006)
Journal Article
The Clonal Inventory of Gene Corrected Hematopoiesis in Three Successful Clinical Gene Therapy Trials
Schwarzwaelder, Kerstin, Schmidt, Manfred, Deichmann, Annette, Howe, Steven J., Ott, Marion G., Stein, Stefan, Siler, Ulrich, Hacein-Bey-Abina, Salima, Glimm, Hanno, Hoelzer, Dieter, Cavazzana-Calvo, Marina, Thrasher, Adrian J., Seger, Reinhard, Grez, Manuel, von Kalle, Christof
Published in Blood (16.11.2007)
Published in Blood (16.11.2007)
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Journal Article
Long-Term Clinical Follow-Up and Safety/Toxicity Analysis in 3 X-CGD Patients Treated by Gene Therapy and Non-Myeloablative Conditioning
Ott, Marion G., Schmidt, Manfred, Stein, Stefan, Schwarzwaelder, Kerstin, Siler, Ulrich, Koehl, Ulrike, Kuehlcke, Klaus, Schilz, Andrea, Kalle, Christoph v., Hoelzer, Dieter, Seger, Reinhard, Grez, Manuel
Published in Blood (16.11.2006)
Published in Blood (16.11.2006)
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Journal Article
Insertional Activation of MDS1/EVI1, PRDM16 and SETBP1 in a Successful Chronic Granulomatous Disease (CGD) Gene Therapy Trial
Schwarzwaelder, Kerstin, Schmidt, Manfred, Deichmann, Annette, Ott, Marion G., Stein, Stefan, Glimm, Hanno, Siler, Ulrich, Hoelzer, Dieter, Seger, Reinhard, Grez, Manuel, von Kalle, Christof
Published in Blood (16.11.2006)
Published in Blood (16.11.2006)
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Journal Article
Phase I/II gene therapy study for X-CGD: Results, lessons and perspectives
Grez, Manuel, Ott, Marion G., Stein, Stefan, Schultze-Strasser, Stephan, Preiss, Carolin, Kramer, Rolf, Schmidt, Manfred, Kunkel, Hana, von Kalle, Christof, Thrasher, Adrian, Hoelzer, Dieter, Seger, Reinhard
Published in Blood cells, molecules, & diseases (01.03.2008)
Published in Blood cells, molecules, & diseases (01.03.2008)
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Journal Article
Phase I/II Gene Therapy Study for Chronic Granulomatous Disease: Results, Lessons and Perspectives
Ott, Marion G., Stein, Stefan, Schultze-Strasser, Stephan, Jauch, Anna, Burwinkel, Barbara, Schlegelberger, Brigitte, Goehring, Gudrun, Preiss, Carolin, Kramer, Rolf, Schwarzwaelder, Kerstin, Koehl, Ulrike, Kunkel, Hanna, Kuehlcke, Klaus, Schilz, Andrea, Schmidt, Manfred, von Kalle, Christof, Trasher, Adrian, Hoelzer, Dieter, Seger, Reinhard, Grez, Manuel
Published in Blood (16.11.2007)
Published in Blood (16.11.2007)
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Journal Article
The gene corrected clonal inventory in clinical gene therapy trials
Schwarzwaelder, Kerstin, Schmidt, Manfred, Deichmann, Annette, Howe, Steven, Ott, Marion G., Stein, Stefan, Siler, Ulrich, Hacein-Bey-Abina, Salima, Glimm, Hanno, Hoelzer, Dieter, Cavazzana-Calvo, Marina, Thrasher, Adrian, Seger, Reinhard, Grez, Manuel, von Kalle, Christof
Published in Blood cells, molecules, & diseases (01.03.2008)
Published in Blood cells, molecules, & diseases (01.03.2008)
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Journal Article
Comparative integration site profile analysis in 5 clinical retroviral gene therapy studies
Deichman, Annette, Schmidt, Manfred, Abina, Salima Hacein-Bey, Cavazzana-Calvo, Marina, Schwarzwaelder, Kerstin, Howe, Steven J., Thrasher, Adrian J., Glimm, Hanno, Ott, Marion G., Hoelzer, Dieter, Grez, Manuel, Seger, Reinhard, Aiuti, Alessandro, von Kalle, Christof
Published in Blood cells, molecules, & diseases (01.03.2008)
Published in Blood cells, molecules, & diseases (01.03.2008)
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Journal Article
Myeloid Expansion after Insertional Activation of MDS1/EVI1, PRDM16 and SETBP1 in a Successful Chronic Granulomatous Gene Therapy Trial
Schwarzwaelder, Kerstin, Schmidt, Manfred, Ott, Marion G., Stein, Stefan, Glimm, Hanno, Deichmann, Annette, Fischer, Marlene, Ball, Claudia, Pilz, Ingo, Schmidt, Sonja, Prinz, Claudia, Fessler, Sylvia, Wissler, Manuela, Klingenberg, Silke, Hoelzer, Dieter, Seger, Reinhard, Grez, Manuel, von Kalle, Christof
Published in Blood (16.11.2005)
Published in Blood (16.11.2005)
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Journal Article
Gene Therapy for X-Linked Chronic Granulomatous Disease
Ott, Marion G., Stein, Stefan, Koehl, Ulrike, Schilz, Andrea, Kuhlcke, Klaus, Schmidt, Manfred, von Kalle, Christof, Hassan, Moustapha, Siler, Ulrich, Hoelzer, Dieter, Seger, Reinhard, Grez, Manuel
Published in Blood (16.11.2004)
Published in Blood (16.11.2004)
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