Search Results - "Ott, Marion G"

Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1 , PRDM16 or SETBP1 by 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

“…Gene transfer into hematopoietic stem cells has been used successfully for correcting lymphoid but not myeloid immunodeficiencies. Here we report on two adults…”
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Venetoclax and idasanutlin in relapsed/refractory AML: a nonrandomized, open-label phase 1b trial by 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

“…•Manageable safety and encouraging preliminary efficacy support extra evaluation of BCL-2/MDM2 inhibition in AML.•IDH1/2 and RUNX1 mutations were associated…”
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Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease by 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

“…Transduced hematopoietic stem cells can benefit patients with X-linked chronic granulomatous disease (a genetic immunodeficiency), but it's not risk free. In…”
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Humoral response to catumaxomab correlates with clinical outcome: Results of the pivotal phase II/III study in patients with malignant ascites by Ott, Marion G., Marmé, Frederik, Moldenhauer, Gerhard, Lindhofer, Horst, Hennig, Michael, Spannagl, Rolf, Essing, Mirko M., Linke, Rolf, Seimetz, Diane

“…The trifunctional antibody catumaxomab is a targeted immunotherapy for the intraperitoneal treatment of malignant ascites. In a Phase II/III trial in cancer…”
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Venetoclax and Cobimetinib in Relapsed/Refractory AML: A Phase 1b Trial by 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

“…•Ven-cobi showed limited efficacy, similar to venetoclax alone, in R/R AML.•Ven-cobi had additional toxicity versus venetoclax alone in patients with R/R…”
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Insertion Sites in Engrafted Cells Cluster Within a Limited Repertoire of Genomic Areas After Gammaretroviral Vector Gene Therapy by 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

“…Vector-associated side effects in clinical gene therapy have provided insights into the molecular mechanisms of hematopoietic regulation in vivo. Surprisingly,…”
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Advances in the treatment of Chronic Granulomatous Disease by gene therapy by Ott, Marion G, Seger, Reinhard, Stein, Stefan, Siler, Ulrich, Hoelzer, Dieter, Grez, Manuel

“…Gene transfer into hematopoietic stem cells has been successfully used to correct immunodeficiencies affecting the lymphoid compartment. However, similar…”
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723. In Vivo Expansion of MDS1/EVI1, PRDM16 and SETBP1 Integration Clones in Successful Chronic Granulomatous Disease (CGD) Gene Therapy Trial by Schwarzwaelder, Kerstin, Schmidt, Manfred, Ott, Marion G., Stein, Stefan, Glimm, Hanno, Deichmann, Annette, Siler, Ulrich, Hoelzer, Dieter, Seger, Reinhard, Grez, Manuel, von Kalle, Christof

“…The potential of gene therapy to cure inherited immunodeficiencies of the lymphoid compartment has been demonstrated in clinical trials of ADA-SCID and…”
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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 by 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

“…▪ Introduction: Elderly pts with R/R AML not eligible for cytotoxic therapy have limited therapeutic options, and dismal outcomes with available therapies. In…”
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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 by 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

“…▪ Introduction Effective therapies for R/R AML remain limited. MEK or MDM2 inhibition can downregulate MCL1, overcoming resistance to BCL2 inhibition…”
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Gene therapy for chronic granulomatous disease by Stein, Stefan, Siler, Ulrich, Ott, Marion G, Seger, Reinhard, Grez, Maunel

“…Chronic granuloniatous disease (CGD) is a rare inherited imnunodeficiency characterized by recurrent, often life threatening bacterial and fungal infections…”
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The Clonal Inventory of Gene Corrected Hematopoiesis in Three Successful Clinical Gene Therapy Trials by 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

“…To enhance the safety and efficacy of future gene therapy trials using integrating vector systems it is necessary to analyse the clonality of the genetically…”
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Long-Term Clinical Follow-Up and Safety/Toxicity Analysis in 3 X-CGD Patients Treated by Gene Therapy and Non-Myeloablative Conditioning by 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

“…Gene transfer into hematopoietic stem cells has been successfully used to correct immunodeficiencies affecting the lymphoid compartment. However, similar…”
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Insertional Activation of MDS1/EVI1, PRDM16 and SETBP1 in a Successful Chronic Granulomatous Disease (CGD) Gene Therapy Trial by Schwarzwaelder, Kerstin, Schmidt, Manfred, Deichmann, Annette, Ott, Marion G., Stein, Stefan, Glimm, Hanno, Siler, Ulrich, Hoelzer, Dieter, Seger, Reinhard, Grez, Manuel, von Kalle, Christof

“…The potential of gene therapy to correct genetic diseases of the lymphoid compartment has been demonstrated in ADA-SCID and X-linked SCID clinical gene therapy…”
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Phase I/II gene therapy study for X-CGD: Results, lessons and perspectives by 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

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Phase I/II Gene Therapy Study for Chronic Granulomatous Disease: Results, Lessons and Perspectives by 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

“…Gene transfer into hematopoietic stem cells has been envisaged as an alternative to stem cell transplantation for the treatment of many genetic diseases of the…”
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The gene corrected clonal inventory in clinical gene therapy trials by 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

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Comparative integration site profile analysis in 5 clinical retroviral gene therapy studies by 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

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Myeloid Expansion after Insertional Activation of MDS1/EVI1, PRDM16 and SETBP1 in a Successful Chronic Granulomatous Gene Therapy Trial by 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

“…Successful gene therapy trials of ADA-SCID and SCID-X1 demonstrated the curative potential of oncoretroviral gene transfer. Integration of the retroviral…”
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Gene Therapy for X-Linked Chronic Granulomatous Disease by 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

“…Chronic Granulomatous Disease (CGD) is a primary immunodeficiency in which phagocytic cells of affected patients have impaired antimicrobial activity due to a…”
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