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Journal Articles

Co-Transplantation of CAR-Gene Engineered Precursor T Cells Leads to Suppressed T Cell Development and Increased NK Cell-Based CAR-Mediated Anti-Leukemic Activity

Marcel Maluski, Jessica Herbst, Vanessa Scholl, Rolf Baumann, MD, Niels Heinz, PhD, Robert Geffers, PhD, Johann Meyer, PhD, Andreas Krueger, PhD, Axel Schambach, MD PhD, Martin G. Sauer

Blood (2017) 130 (Supplement 1): 1916.

https://doi.org/10.1182/blood.V130.Suppl_1.1916.1916

Published: 2017

Journal Articles

Generation of Functional Regulatory T Cells By FOXP3 Gene Transfer into CD4 T Cells from Scurfy Mice and IPEX Patients

Marianne Delville, MD, Emmanuelle Six, PhD, Florence Bellier, Nelly Sigrist, David Zemmour, PhD, Marie-André Bessard, MD, Fabienne Mazerolles, PhD, David Gross, PhD, Axel Schambach, MD PhD, Frederic Rieux-Laucat, PhD, Christoph Benoist, MD PhD, Isabelle Andre-Schmutz, Marina Cavazzana, MD PhD

Blood (2016) 128 (22): 2526.

https://doi.org/10.1182/blood.V128.22.2526.2526

Published: 2016

Journal Articles

Preclinical Development of Gene Therapy for X-Linked Severe Combined Immunodeficiency (SCID-X1)

Valentina Poletti, PhD, Sabine Charrier, PhD, Samia Martin, Bernard Gjata, Alban Vignaud, PhD, Fang Zhang, PhD, Karen Buckland, PhD, Michael Rothe, PhD, Axel Schambach, MD PhD, Sung-Yun Pai, MD, David A. Williams, MD, H. Bobby Gaspar, MD PhD, Adrian J Thrasher, MD PhD, Fulvio Mavilio, PhD

Blood (2016) 128 (22): 4705.

https://doi.org/10.1182/blood.V128.22.4705.4705

Published: 2016

Journal Articles

New Molecular Surrogate Assay for Genotoxicity Assessment of Gene Therapy Vectors (SAGA)

Adrian Schwarzer, MD PhD, Steven R Talbot, PhD, Oliver Dittrich-Breiholz, PhD, Adrian J Thrasher, MD PhD, Bobby Gaspar, MD, Ute Modlich, Christopher Baum, MD, Axel Schambach, MD PhD, Michael Rothe, PhD

Blood (2016) 128 (22): 4710.

https://doi.org/10.1182/blood.V128.22.4710.4710

Published: 2016

Journal Articles

Innovative Hematopoietic Gene-Therapy Concepts for Hereditary Pulmonary Alveolar Proteinosis Utilizing Hematopoietic Stem Cell Derived Macrophages

Nico Lachmann, PhD, Christine Happle, MD, Takuji Suzuki, MD, Miriam Hetzel, Kevin A. Link, PhD, Adele Mucci, Paritha Arumugam, PhD, Jens Bankstahl, PhD, Dagmar Dilloo, MD, Punam Malik, MD, Carolyn Lutzko, PhD, Axel Schambach, MD PhD, Bruce Colston Trapnell, MD, Gesine Hansen, MD, Thomas Moritz, MD

Blood (2015) 126 (23): 4417.

https://doi.org/10.1182/blood.V126.23.4417.4417

Published: 2015

Journal Articles

Targeting Rac-Gtpases Reverses Stroma-Induced Resistance to Notch and mTOR-Inhibition in Acute T-Cell Leukemia

Adrian Schwarzer, MD PhD, Martin May, Harald Genth, Zhixiong Li, MD, Christopher Baum, MD, Axel Schambach, MD PhD

Blood (2015) 126 (23): 1326.

https://doi.org/10.1182/blood.V126.23.1326.1326

Published: 2015

Journal Articles

Dynamic Telomere Shortening and Chromosomal Instability in Irradiated CD34+ Cells Transduced with TP53 Hotspot Mutations R175H, R248W and R249S

Azam Salari, Kathrin Thomay, Dr. rer. nat., Andrea Schienke, Maike Hagedorn, Juliane Ebersold, Andreas Krueger, PhD Prof., Axel Schambach, MD PhD, Brigitte Schlegelberger, MD Prof., Gudrun Göhring, MD

Blood (2015) 126 (23): 4832.

https://doi.org/10.1182/blood.V126.23.4832.4832

Published: 2015

Journal Articles

No Observation of Chromosomal Instability after Transplantation of RPS14- and TP53-Modified Human HSCs in NSG/NSGS Mice

Kathrin Thomay, Dr. rer. nat., Azam Salari, Beate Vajen, Dr. rer. nat., Andrea Schienke, Maike Hagedorn, Juliane Ebersold, Hans Heinrich Kreipe, MD, Andreas Krueger, PhD Prof., Axel Schambach, MD PhD, Brigitte Schlegelberger, MD Prof., Gudrun Göhring, MD

Blood (2015) 126 (23): 4797.

https://doi.org/10.1182/blood.V126.23.4797.4797

Published: 2015

Journal Articles

Investigating Synthetic Lethality Between mTOR Hyperactivation and Cap-Dependent mRNA Translation In a Receptor Tyrosine Kinase Driven Model Of Acute T-Cell Leukemia

Adrian Schwarzer, MD PhD, Helmut Holtmann, PhD, Celina Schauerte, M.Sc., Nomme Benn, Jan Völkel, Sabine Knöß, Johannes Zuber, MD, Zhixiong Li, MD, Axel Schambach, MD PhD, Christopher Baum, MD

Blood (2013) 122 (21): 3913.

https://doi.org/10.1182/blood.V122.21.3913.3913

Published: 2013

Journal Articles

Immune Reconstitution and Preliminary Safety Analysis Of 9 Patients Treated With Somatic Gene Therapy For X-Linked Severe Combined Immunodeficiency (SCID-X1) With a Self-Inactivating Gammaretroviral Vector

Blood (2013) 122 (21): 715.

https://doi.org/10.1182/blood.V122.21.715.715

Published: 2013

Journal Articles

Induced Pluripotent Stem Cells From a Patient with Reticular Dysgenesis Recapitulate Defective Myelopoiesis in-Vitro: A Disease Model to Enhance Our Understanding of a Rare Disease.

Katja G. Weinacht, MD, PhD, Kerstin Felgentreff, MD, Alex Devine, MS, Axel Schambach, MD PhD, Waleed Al-herz, MD, Luigi Daniele Notarangelo, MD

Blood (2012) 120 (21): 2142.

https://doi.org/10.1182/blood.V120.21.2142.2142

Published: 2012

Journal Articles

Gene Therapy Corrects the Lethal Bone Marrow Failure in a Mouse Model for RPS19-Deficient Diamond-Blackfan Anemia

Pekka Jaako, Shubhranshu Debnath, Karin Olsson, Axel Schambach, MD, PhD, Christopher Baum, MD, Johan Flygare, MD, PhD, Stefan Karlsson, MD, PhD

Blood (2012) 120 (21): 513.

https://doi.org/10.1182/blood.V120.21.513.513

Published: 2012

Journal Articles

the miR-99∼125 P olycistrons Promote Leukemogenesis in a Cell-Context Dependent Manner by Shifting the Balance Between TGFβ- and Wnt-Signaling

Stephan Emmrich, Mareike Rasche, Jennifer Schöning, Zhe Li, PhD, Axel Schambach, MD PhD, Dirk Reinhardt, MD, Jan-Henning Klusmann, MD

Blood (2012) 120 (21): 109.

https://doi.org/10.1182/blood.V120.21.109.109

Published: 2012

Journal Articles

Somatic Gene Therapy for X-Linked Severe Combined Immunodeficiency Using a Self-Inactivating Modified Gammaretroviral Vector Results in An Improved Preclinical Safety Profile and Early Clinical Efficacy in a Human Patient

Blood (2011) 118 (21): 164.

https://doi.org/10.1182/blood.V118.21.164.164

Published: 2011

Journal Articles

Gene-Correction Rescues Reprogramming of Fanconi Anemia Fibroblasts and Enables Hematopoietic Differentiation of FA Induced Pluripotent Stem Cells in Vitro and In Vivo

Lars Mueller, MD, Michael D. Milsom, PhD, Chad Harris, Rutesh Vyas, Kristina Brumme, Kalindi Parmar, PhD, Axel Schambach, MD PhD, Elke Grassman, Ph.D., In-Hyun Park, PhD, London Wendy, PhD, Kelly Strait, Thorsten Schlaeger, Ph.D, Alexander L. Devine, Alan D. D'Andrea, MD, George Q. Daley, MD, PhD, David A. Williams, MD

Blood (2011) 118 (21): 672.

https://doi.org/10.1182/blood.V118.21.672.672

Published: 2011

Journal Articles

Use of the in Vitro Immortalization Assay to Quantify the Impact of Integration Spectrum and Vector Design on Insertional Mutagenesis

Ute Modlich, DVM, PhD, Julia Sürth, Daniela Zychlinski, PhD, Johann Meyer, PhD, Christian Brendel, Manuel Grez, PhD, Paritha Arumugam, PhD, Punam Malik, MD, Elke Grassman, PhD, Axel Schambach, MD PhD, Christopher Baum, MD

Blood (2011) 118 (21): 3123.

https://doi.org/10.1182/blood.V118.21.3123.3123

Published: 2011

Journal Articles

Telomere Attrition and Chromosomal Instability During Long-Term Cultivation of Hematopoietic Stem Cells,

Kathrin Lange, Dipl. Biochem., Beate Kuhlmann, PhD, Andrea Schienke, Axel Schambach, MD PhD, Ute Modlich, DVM, PhD, Christopher Baum, MD, Brigitte Schlegelberger, M.D., Gudrun Göhring, MD

Blood (2011) 118 (21): 4001.

https://doi.org/10.1182/blood.V118.21.4001.4001

Published: 2011

Journal Articles

Deciphering the Role of Mir-99∼125 clusters in the Hematopoietic System

Stephan Emmrich, Kerstin Henke, Zhe Li, PhD, Jennifer Schöning, Axel Schambach, MD PhD, Dirk Reinhardt, MD, PhD, Jan-Henning Klusmann, MD

Blood (2011) 118 (21): 213.

https://doi.org/10.1182/blood.V118.21.213.213

Published: 2011

Journal Articles

Doxycycline Regulatable Expression of Cytidine Deaminase Mediates Myeloprotection and Avoids Lymphotoxicity in a Murine Transplant Model

Nico Lachmann, Sebastian Brennig, Nils Pfaff, Heiko Schermeier, Axel Schambach, MD PhD, Christopher Baum, MD, Thomas Moritz, MD

Blood (2011) 118 (21): 2054.

https://doi.org/10.1182/blood.V118.21.2054.2054

Published: 2011

Journal Articles

Evidence for Cooperation of Receptor Tyrosine Kinases and Activating NOTCH Mutations to Hyperactivate mTOR in T-Cell Leukemia: A Rationale Basis for Targeted Therapy

Adrian Schwarzer, MD, Johann Meyer, PhD, Martijn Brugman, PhD, Axel Schambach, MD PhD, Martin Stanulla, MD, MSc, Martin Schrappe, MD, Doris Steinemann, PhD, Brigitte Schlegelberger, M.D., Christopher Baum, MD, Zhixiong Li, MD

Blood (2011) 118 (21): 1381.

https://doi.org/10.1182/blood.V118.21.1381.1381

Published: 2011

NARROW

Co-Transplantation of CAR-Gene Engineered Precursor T Cells Leads to Suppressed T Cell Development and Increased NK Cell-Based CAR-Mediated Anti-Leukemic Activity

Generation of Functional Regulatory T Cells By FOXP3 Gene Transfer into CD4 T Cells from Scurfy Mice and IPEX Patients

Preclinical Development of Gene Therapy for X-Linked Severe Combined Immunodeficiency (SCID-X1)

New Molecular Surrogate Assay for Genotoxicity Assessment of Gene Therapy Vectors (SAGA)

Innovative Hematopoietic Gene-Therapy Concepts for Hereditary Pulmonary Alveolar Proteinosis Utilizing Hematopoietic Stem Cell Derived Macrophages

Targeting Rac-Gtpases Reverses Stroma-Induced Resistance to Notch and mTOR-Inhibition in Acute T-Cell Leukemia

Dynamic Telomere Shortening and Chromosomal Instability in Irradiated CD34+ Cells Transduced with TP53 Hotspot Mutations R175H, R248W and R249S

No Observation of Chromosomal Instability after Transplantation of RPS14- and TP53-Modified Human HSCs in NSG/NSGS Mice

Investigating Synthetic Lethality Between mTOR Hyperactivation and Cap-Dependent mRNA Translation In a Receptor Tyrosine Kinase Driven Model Of Acute T-Cell Leukemia

Immune Reconstitution and Preliminary Safety Analysis Of 9 Patients Treated With Somatic Gene Therapy For X-Linked Severe Combined Immunodeficiency (SCID-X1) With a Self-Inactivating Gammaretroviral Vector

Induced Pluripotent Stem Cells From a Patient with Reticular Dysgenesis Recapitulate Defective Myelopoiesis in-Vitro: A Disease Model to Enhance Our Understanding of a Rare Disease.

Gene Therapy Corrects the Lethal Bone Marrow Failure in a Mouse Model for RPS19-Deficient Diamond-Blackfan Anemia

the miR-99∼125 P olycistrons Promote Leukemogenesis in a Cell-Context Dependent Manner by Shifting the Balance Between TGFβ- and Wnt-Signaling

Somatic Gene Therapy for X-Linked Severe Combined Immunodeficiency Using a Self-Inactivating Modified Gammaretroviral Vector Results in An Improved Preclinical Safety Profile and Early Clinical Efficacy in a Human Patient

Gene-Correction Rescues Reprogramming of Fanconi Anemia Fibroblasts and Enables Hematopoietic Differentiation of FA Induced Pluripotent Stem Cells in Vitro and In Vivo

Use of the in Vitro Immortalization Assay to Quantify the Impact of Integration Spectrum and Vector Design on Insertional Mutagenesis

Telomere Attrition and Chromosomal Instability During Long-Term Cultivation of Hematopoietic Stem Cells,

Deciphering the Role of Mir-99∼125 clusters in the Hematopoietic System

Doxycycline Regulatable Expression of Cytidine Deaminase Mediates Myeloprotection and Avoids Lymphotoxicity in a Murine Transplant Model

Evidence for Cooperation of Receptor Tyrosine Kinases and Activating NOTCH Mutations to Hyperactivate mTOR in T-Cell Leukemia: A Rationale Basis for Targeted Therapy

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American Society of Hematology

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American Society of Hematology

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