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Journal Articles

Lentiviral-Mediated Gene Therapy for Patients with Fanconi Anemia [Group a]: Updated Results from Global RP-L102 Clinical Trials

Blood (2024) 144 (Supplement 1): 7463.

https://doi.org/10.1182/blood-2024-194638

Published: 2024

Journal Articles

Single-Cell-Multiomics Demonstrates Molecular Efficacy of a Clinical Lentiviral Vector for Gene Therapy of RPS19-Deficient Diamond-Blackfan Anemia

Sofia Freiman, Jun Chen, Johan Flygare, Chayenne Kampman, Karin Pike-Overzet, Alexander Mohseny, Teng-Cheong Ha, Mathias Lidgren, Shamit Soneji, Stefan Karlsson, Dagmar Pospíšilová, Frank Staal, Axel Schambach, Johan Flygare

Blood (2023) 142 (Supplement 1): 943.

https://doi.org/10.1182/blood-2023-185802

Published: 2023

Journal Articles

In Vivo Modeling of T-Cell Acute Lymphoblastic Leukemia Reveals Synergistic Oncogenic Pathways and Bcl11b Haploinsufficiency As a Potential Therapeutic Vulnerability

Maximilian Schinke, Marc-Jens Kleppa, Dirk Heckl, Michael Heuser, Axel Schambach, Adrian Schwarzer

Blood (2023) 142 (Supplement 1): 4149.

https://doi.org/10.1182/blood-2023-190515

Published: 2023

Journal Articles

Bcl11b -Edited Lymphoid Progenitors for the Generation of CAR-Engineered Natural Killer-like Cells with Potent Anti-Leukemic Activity

Franziska Baatz, Jessica Herbst, Johann Meyer, Axel Schambach, Tobias Maetzig, Michael Hust, Martin G. Sauer

Blood (2022) 140 (Supplement 1): 2862–2863.

https://doi.org/10.1182/blood-2022-157663

Published: 2022

Journal Articles

Long-Term Outcome of Gene Therapy for X-Linked Severe Combined Immunodeficiency (SCID-X1) Using an Enhancer-Deleted Self-Inactivating Gammaretroviral Vector

Blood (2022) 140 (Supplement 1): 10643–10645.

https://doi.org/10.1182/blood-2022-159768

Published: 2022

Journal Articles

Single and Combinational Multiplex Base-Edited 'Universal' CAR T Cells in a Humanised Model of Primary CD7+CD33+ AML

Arnold Kloos, Christos Georgiadis, Annie Etuk, Soragia Athina Gkazi, Farhatullah Syed, Toni Braybrook, Hong Zhan, Renuka Kadirkamanathan, Nadine Kattre, Kerstin Görlich, Thomas Fangmann, Axel Schambach, Martin G. Sauer, Michael Heuser, Waseem Qasim

Blood (2022) 140 (Supplement 1): 7425–7426.

https://doi.org/10.1182/blood-2022-168719

Published: 2022

Journal Articles

Lentiviral Gene Therapy with Low Dose Conditioning for X-Linked SCID Results in Complete Immune Reconstitution and No Evidence of Clonal Expansion

Blood (2022) 140 (Supplement 1): 7770–7771.

https://doi.org/10.1182/blood-2022-159979

Published: 2022

Journal Articles

A combination of cyclophosphamide and interleukin-2 allows CD4 + T cells converted to Tregs to control scurfy syndrome

Blood (2021) 137 (17): 2326–2336.

https://doi.org/10.1182/blood.2020009187

Published: 2021

Includes: Supplemental data

Journal Articles

Hematopoietic stem cell gene therapy for IFNγR1 deficiency protects mice from mycobacterial infections

Blood (2018) 131 (5): 533–545.

https://doi.org/10.1182/blood-2017-10-812859

Published: 2018

Includes: Multimedia, Supplemental data

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

CSF-1–induced Src signaling can instruct monocytic lineage choice

Max Endele, Dirk Loeffler, Konstantinos D. Kokkaliaris, Oliver Hilsenbeck, Stavroula Skylaki, Philipp S. Hoppe, Axel Schambach, E. Richard Stanley, Timm Schroeder

Blood (2017) 129 (12): 1691–1701.

https://doi.org/10.1182/blood-2016-05-714329

Published: 2017

Includes: Multimedia, Supplemental data

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

Limited niche availability suppresses murine intrathymic dendritic-cell development from noncommitted progenitors

Marcin Łyszkiewicz, Natalia Ziętara, Lisa Föhse, Jacek Puchałka, Jana Diestelhorst, Katrin Witzlau, Immo Prinz, Axel Schambach, Andreas Krueger

Blood (2015) 125 (3): 457–464.

https://doi.org/10.1182/blood-2014-07-592667

Published: 2015

Includes: Supplemental data

Journal Articles

Timely Controlled T Cell Receptor Expression of Genetically Engineered Precursor T Cells Requires Early Transgene Induction for Leukemia Control after Hematopoietic Stem Cell Transplantation

Sayed Shahabuddin Hoseini, Martin Hapke, Jessica Herbst, Dirk Wedekind, PhD, Rolf Baumann, PhD, Andreas Krueger, Niels Heinz, Bernhard Schiedlmeier, PhD, Dario Vignali, PhD, Marcel R.M. van den Brink, MD PhD, Axel Schambach, MDPhD, Bruce R. Blazar, MD, Martin G. Sauer, MD

Blood (2014) 124 (21): 657.

https://doi.org/10.1182/blood.V124.21.657.657

Published: 2014

NARROW

Lentiviral-Mediated Gene Therapy for Patients with Fanconi Anemia [Group a]: Updated Results from Global RP-L102 Clinical Trials

Single-Cell-Multiomics Demonstrates Molecular Efficacy of a Clinical Lentiviral Vector for Gene Therapy of RPS19-Deficient Diamond-Blackfan Anemia

In Vivo Modeling of T-Cell Acute Lymphoblastic Leukemia Reveals Synergistic Oncogenic Pathways and Bcl11b Haploinsufficiency As a Potential Therapeutic Vulnerability

Bcl11b -Edited Lymphoid Progenitors for the Generation of CAR-Engineered Natural Killer-like Cells with Potent Anti-Leukemic Activity

Long-Term Outcome of Gene Therapy for X-Linked Severe Combined Immunodeficiency (SCID-X1) Using an Enhancer-Deleted Self-Inactivating Gammaretroviral Vector

Single and Combinational Multiplex Base-Edited 'Universal' CAR T Cells in a Humanised Model of Primary CD7+CD33+ AML

Lentiviral Gene Therapy with Low Dose Conditioning for X-Linked SCID Results in Complete Immune Reconstitution and No Evidence of Clonal Expansion

A combination of cyclophosphamide and interleukin-2 allows CD4 + T cells converted to Tregs to control scurfy syndrome

Hematopoietic stem cell gene therapy for IFNγR1 deficiency protects mice from mycobacterial infections

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

CSF-1–induced Src signaling can instruct monocytic lineage choice

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

Limited niche availability suppresses murine intrathymic dendritic-cell development from noncommitted progenitors

Timely Controlled T Cell Receptor Expression of Genetically Engineered Precursor T Cells Requires Early Transgene Induction for Leukemia Control after Hematopoietic Stem Cell Transplantation

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American Society of Hematology

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ASH Publications

American Society of Hematology

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