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Journal Articles

Favorable Outcomes in Pediatric Patients in the Phase 3 Hgb-207 (Northstar-2) and Hgb-212 (Northstar-3) Studies of Betibeglogene Autotemcel Gene Therapy for the Treatment of Transfusion-Dependent β-Thalassemia

Alexis A. Thompson, MD, Janet L. Kwiatkowski, MD MSCE, John B. Porter, MA, MD, FRCP, FRCPath, Suradej Hongeng, MD, Evangelia Yannaki, MD, Andreas E. Kulozik, MD PhD, Martin G. Sauer, MD, Adrian J. Thrasher, MBBS, PhD FMedSci, Isabelle Thuret, MD, Ashutosh Lal, MD, Ruiting Guo, MS, Weijian Liu, PhD, Richard A. Colvin, MD PhD, Mark C. Walters, MD, Franco Locatelli, MD PhD

Blood (2020) 136 (Supplement 1): 52–54.

https://doi.org/10.1182/blood-2020-135857

Published: 2020

Journal Articles

Co-Targeting of CD38 and CD47 in T Cell Acute Lymphoblastic Leukemia

Fotini Vogiatzi, PhD, Kristina Müller, Dorothee Winterberg, Thies Rösner, PhD, Lennart Lenk, PhD, Gunnar Cario, MD PhD, Martin Schrappe, MDPhD, Andreas E. Kulozik, MD PhD, Beat Bornhauser, Jean-Pierre Bourquin, MD PhD, Thomas Valerius, MD, Matthias Peipp, PhD, Christian Kellner, PhD, Denis Martin Schewe, MD

Blood (2020) 136 (Supplement 1): 39–40.

https://doi.org/10.1182/blood-2020-140270

Published: 2020

Journal Articles

Response of Patients with Transfusion-Dependent β-Thalassemia (TDT) to Betibeglogene Autotemcel (beti-cel; LentiGlobin for β-Thalassemia) Gene Therapy Based on HBB Genotype and Disease Genetic Modifiers

Mark C. Walters, MD, David H.K. Chui, MD FRCPC, John J Farrell, PhD, Ashutosh Lal, MD, Franco Locatelli, MD PhD, Janet L. Kwiatkowski, MD MSCE, John B. Porter, MA, MD, FRCP, FRCPath, Martin G. Sauer, MD, Isabelle Thuret, MD, Suradej Hongeng, MD, Andreas E. Kulozik, MD PhD, Adrian J. Thrasher, MBBS, PhD FMedSci, Evangelia Yannaki, MD, Julia Yang, Dustin Whitney, PhD, Alexandria Petrusich, Richard A. Colvin, MD PhD, Alexis A. Thompson, MD

Blood (2020) 136 (Supplement 1): 1–3.

https://doi.org/10.1182/blood-2020-137642

Published: 2020

Journal Articles

In Vitro Drug Response Profiling in BCP- and T-ALL Primary Samples Adds a Robust Functional Layer Enabling Optimized Guidance of Individualized Therapy in Relapsed and Refractory Pediatric Acute Leukemia Patients

Blood (2020) 136 (Supplement 1): 15–16.

https://doi.org/10.1182/blood-2020-142314

Published: 2020

Journal Articles

Northstar-3: Interim Results from a Phase 3 Study Evaluating Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Either a β0 or IVS-I-110 Mutation at Both Alleles of the HBB Gene

Ashutosh Lal, MD, Franco Locatelli, MD PhD, Janet L. Kwiatkowski, MD MSCE, Andreas E. Kulozik, MD PhD, Evangelia Yannaki, MD, John B. Porter, MA MDFRCP FRCPath, Isabelle Thuret, MD, Martin G. Sauer, MD, Heidi Elliot, Ying Chen, PhD, Richard A. Colvin, MD PhD, Alexis A. Thompson, MD MPH

Blood (2019) 134 (Supplement_1): 815.

https://doi.org/10.1182/blood-2019-128482

Published: 2019

Journal Articles

Pediatric T-ALLs Developing into a Type 2 Relapse Originate from Cells That Carry the Potential of Variable Maturation into Subclones with Distinct Chromatin Landscapes

Blood (2018) 132 (Supplement 1): 1545.

https://doi.org/10.1182/blood-2018-99-118255

Published: 2018

Journal Articles

Longitudinal Multilevel Omic Analysis of Pediatric T-ALL Reveals Distinct Mechanisms for Disease Progression in Type 1 and in Type 2 Relapses

Blood (2018) 132 (Supplement 1): 2826.

https://doi.org/10.1182/blood-2018-99-117978

Published: 2018

Journal Articles

Lentiglobin Gene Therapy for Patients with Transfusion-Dependent β-Thalassemia (TDT): Results from the Phase 3 Northstar-2 and Northstar-3 Studies

Franco Locatelli, Mark C. Walters, MD, Janet L. Kwiatkowski, MD, John Porter, MA MDFRCP FRCPath, Martin G. Sauer, MD, Isabelle Thuret, MD, Suradej Hongeng, MD, Andreas E. Kulozik, MD PhD, Ashutosh Lal, MD, Adrian J. Thrasher, MD PhD FMedSci, Evangelia Yannaki, MD, Heidi Elliot, Ge Tao, PhD, Mohammed Asmal, MD PhD, Alexis A. Thompson, MD MPH

Blood (2018) 132 (Supplement 1): 1025.

https://doi.org/10.1182/blood-2018-99-112667

Published: 2018

Journal Articles

Response to Front-Line Imatinib Treatment in Children and Adolescents with CML - Data from a Large Pediatric Cohort

Blood (2017) 130 (Supplement 1): 898.

https://doi.org/10.1182/blood.V130.Suppl_1.898.898

Published: 2017

Journal Articles

PDX Models Recapitulate Genetics and Epigenetics of Pediatric T-Cell Leukemia

Blood (2017) 130 (Supplement 1): 2723.

https://doi.org/10.1182/blood.V130.Suppl_1.2723.2723

Published: 2017

Journal Articles

Relapsing Pediatric T-Cell Acute Lymphoblastic Leukemia Downregulates T-Cell Properties and Upregulates Cell Adhesion

Blood (2017) 130 (Supplement 1): 2726.

https://doi.org/10.1182/blood.V130.Suppl_1.2726.2726

Published: 2017

Journal Articles

PTEN Alterations Are Related to Unfavorable Prognosis in Children with T-Cell Acute Lymphoblastic Leukemia Treated According to ALL IC-BFM Protocols

Bronisława Szarzyńska-Zawadzka, Joachim B. Kunz, MD, Maria Kosmalska, Katarzyna Zdon, Przemysław Biecek, PhD, Obul Reddy Bandapalli, PhD, Sędek Łukasz, PhD, Jerzy R. Kowalczyk, Prof, MD PhD, Tomasz Szczepański, MD PhD, Andreas E. Kulozik, MD PhD, Michał Witt, MD PhD, Malgorzata Dawidowska, PhD

Blood (2017) 130 (Supplement 1): 1446.

https://doi.org/10.1182/blood.V130.Suppl_1.1446.1446

Published: 2017

Journal Articles

MAP3K7 Is Recurrently Deleted in Pediatric T-Lymphoblastic Leukemia and Affects Cell Proliferation Independently of NF-κB

David M. Cordas dos Santos, Juliane Eilers, Alfonso Vizcaino, Elena Orlova, Martin Zimmermann, PhD, Martin Stanulla, MD PhD, Martin Schrappe, MD PhD, Kathleen Börner, PhD, Dirk Grimm, PhD, Martina Muckenthaler, Andreas E. Kulozik, MD PhD, Joachim B. Kunz, MD

Blood (2017) 130 (Supplement 1): 1447.

https://doi.org/10.1182/blood.V130.Suppl_1.1447.1447

Published: 2017

Journal Articles

The Combination of MRD and Copy Number Alterations (CNAs) Defines an Ultra-High Risk Group of Children with Primary T-Lymphoblastic Leukemia (T-ALL)

Joachim B. Kunz, MD, Julia Seemann, Martin Zimmermann, PhD, Paulina Richter-Pechanska, Obul Reddy Bandapalli, PhD, Elena Orlova, Gunnar Cario, Martin Stanulla, MD, Martin Schrappe, MD PhD, Marketa Zaliova, Jan Trka, MD PhD, Renate Panzer-Grümayer, MD, Georg Mann, MD, Martina U. Muckenthaler, PhD, Jan O. Korbel, PhD, Andreas E Kulozik, MD PhD

Blood (2016) 128 (22): 2911.

https://doi.org/10.1182/blood.V128.22.2911.2911

Published: 2016

Journal Articles

Identification of an Ultra High-Risk and Targetable Molecular Signature in Relapsed Pediatric T-ALL

Paulina Richter-Pechanska, PhD, Joachim B. Kunz, MD, Jana Hof, Martin Zimmermann, PhD, Tobias Rausch, PhD, Obul Reddy Bandapalli, PhD, Elena Orlova, Greta Scapinello, Judit Sagi, Martin Stanulla, MD, Martin Schrappe, MD PhD, Renate Kirschner-Schwabe, PhD, Cornelia Eckert, PhD, Vladimir Benes, PhD, Jan O. Korbel, PhD, Martina U. Muckenthaler, PhD, Andreas E Kulozik, MD PhD

Blood (2016) 128 (22): 1084.

https://doi.org/10.1182/blood.V128.22.1084.1084

Published: 2016

Journal Articles

Drug Response Profiling to Identify Selective Pharmacological Activity in Drug Resistant ALL

Viktoras Frismantas, MSc, Maria Pamela Dobay, PhD, Anna Rinaldi, PhD, Kunz Joachim, MD, Blerim Marovca, Peter Horvath, PhD, Mauro Delorenzi, PhD, Gunnar Cario, MD, Martin Schrappe, MD, Martin Stanulla, MD, Andreas E. Kulozik, MD PhD, Martina U. Muckenthaler, PhD, Arend Stackelberg, MD, Cornelia Eckert, Thomas Radimerski, PhD, Beat Bornhauser, PhD, Jean-Pierre Bourquin, MD PhD

Blood (2015) 126 (23): 2532.

https://doi.org/10.1182/blood.V126.23.2532.2532

Published: 2015

Journal Articles

The Predominant Activation of Alternative Signaling Pathways Explains the Differential Propensity of Different c-Mpl Gain-of-Function Mutations to Promote Myeloproliferative Malignancies

Clemens Stockklausner, MD, Christin Maria Duffert, Nicole Dickemann, Anne Christine Klotter, Isabelle Nadine Kuhlee, Carolin Kerber, Andreas E. Kulozik, MD PhD

Blood (2014) 124 (21): 1578.

https://doi.org/10.1182/blood.V124.21.1578.1578

Published: 2014

Journal Articles

Whole Exome Sequencing Identifies Novel Lyst-Missense Mutations In Incomplete Childhood Chediak-Higashi-Syndrome Presenting As Hemphagocytic Lymphohistiocytosis (HLH)

Joachim Kunz, MD, Tobias Rausch, PhD, Obul R Bandapalli, PhD, Adrian M Stuetz, PhD, Johann Greil, MD, Ingrid Hausser, MD, Jan Korbel, PhD, Andreas E Kulozik, MD, PhD

Blood (2013) 122 (21): 3479.

https://doi.org/10.1182/blood.V122.21.3479.3479

Published: 2013

Journal Articles

Whole Exome Sequencing In Relapsed Pediatric T-ALL: Progression Into Relapse Is Characterized By An Increased Number Of Somatic Mutations and a Conservation Of Mutations In Leukemogenic Driver Genes

Joachim Kunz, MD, Tobias Rausch, PhD, Obul R Bandapalli, PhD, Martina U. Muckenthaler, PhD, Adrian M Stuetz, PhD, Martin Stanulla, MD, MSc, Martin Schrappe, MD, Rupert Handgretinger, Smadar Avigad, PhD, Jan Korbel, PhD, Andreas E. Kulozik, MD, PhD

Blood (2013) 122 (21): 228.

https://doi.org/10.1182/blood.V122.21.228.228

Published: 2013

Journal Articles

Whole-Exome Sequencing Links CARD11 Inactivation with SCID

Johann Greil, MD, Tobias Rausch, PhD, Thomas Giese, MD, Obul Reddy Bandapalli, PhD, Volker Daniel, MD, PhD, Isabelle Bekeredjian-Ding, PhD, Adrian M Stuetz, PhD, Christoph Drees, PhD, Susanne Roth, MD, Jürgen Ruland, MD, Jan Korbel, PhD, Andreas E Kulozik, MD, PhD

Blood (2012) 120 (21): 258.

https://doi.org/10.1182/blood.V120.21.258.258

Published: 2012

NARROW

Favorable Outcomes in Pediatric Patients in the Phase 3 Hgb-207 (Northstar-2) and Hgb-212 (Northstar-3) Studies of Betibeglogene Autotemcel Gene Therapy for the Treatment of Transfusion-Dependent β-Thalassemia

Co-Targeting of CD38 and CD47 in T Cell Acute Lymphoblastic Leukemia

Response of Patients with Transfusion-Dependent β-Thalassemia (TDT) to Betibeglogene Autotemcel (beti-cel; LentiGlobin for β-Thalassemia) Gene Therapy Based on HBB Genotype and Disease Genetic Modifiers

In Vitro Drug Response Profiling in BCP- and T-ALL Primary Samples Adds a Robust Functional Layer Enabling Optimized Guidance of Individualized Therapy in Relapsed and Refractory Pediatric Acute Leukemia Patients

Northstar-3: Interim Results from a Phase 3 Study Evaluating Lentiglobin Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia and Either a β0 or IVS-I-110 Mutation at Both Alleles of the HBB Gene

Pediatric T-ALLs Developing into a Type 2 Relapse Originate from Cells That Carry the Potential of Variable Maturation into Subclones with Distinct Chromatin Landscapes

Longitudinal Multilevel Omic Analysis of Pediatric T-ALL Reveals Distinct Mechanisms for Disease Progression in Type 1 and in Type 2 Relapses

Lentiglobin Gene Therapy for Patients with Transfusion-Dependent β-Thalassemia (TDT): Results from the Phase 3 Northstar-2 and Northstar-3 Studies

Response to Front-Line Imatinib Treatment in Children and Adolescents with CML - Data from a Large Pediatric Cohort

PDX Models Recapitulate Genetics and Epigenetics of Pediatric T-Cell Leukemia

Relapsing Pediatric T-Cell Acute Lymphoblastic Leukemia Downregulates T-Cell Properties and Upregulates Cell Adhesion

PTEN Alterations Are Related to Unfavorable Prognosis in Children with T-Cell Acute Lymphoblastic Leukemia Treated According to ALL IC-BFM Protocols

MAP3K7 Is Recurrently Deleted in Pediatric T-Lymphoblastic Leukemia and Affects Cell Proliferation Independently of NF-κB

The Combination of MRD and Copy Number Alterations (CNAs) Defines an Ultra-High Risk Group of Children with Primary T-Lymphoblastic Leukemia (T-ALL)

Identification of an Ultra High-Risk and Targetable Molecular Signature in Relapsed Pediatric T-ALL

Drug Response Profiling to Identify Selective Pharmacological Activity in Drug Resistant ALL

The Predominant Activation of Alternative Signaling Pathways Explains the Differential Propensity of Different c-Mpl Gain-of-Function Mutations to Promote Myeloproliferative Malignancies

Whole Exome Sequencing Identifies Novel Lyst-Missense Mutations In Incomplete Childhood Chediak-Higashi-Syndrome Presenting As Hemphagocytic Lymphohistiocytosis (HLH)

Whole Exome Sequencing In Relapsed Pediatric T-ALL: Progression Into Relapse Is Characterized By An Increased Number Of Somatic Mutations and a Conservation Of Mutations In Leukemogenic Driver Genes

Whole-Exome Sequencing Links CARD11 Inactivation with SCID

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