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Journal Articles

Clinical Outcomes up to 3 Years Following Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar Hgb-204 Study

Janet L. Kwiatkowski, MD, Alexis A Thompson, MD MPH, John Rasko, MBBS, PhD, Suradej Hongeng, MD, Gary J. Schiller, MD, Usanarat Anurathapan, Marina Cavazzana, MD PhD, Phoebe Joy Ho, Christof von Kalle, MD, Morris Kletzel, MD MBA, Philippe Leboulch, MD, Elliott Vichinsky, MD, Briana Deary, Mohammed Asmal, MD PhD, Mark C. Walters, MD

Blood (2017) 130 (Supplement 1): 360.

https://doi.org/10.1182/blood.V130.Suppl_1.360.360

Published: 2017

Journal Articles

Interim Results from a Phase 1/2 Clinical Study of Lentiglobin Gene Therapy for Severe Sickle Cell Disease

Julie Kanter, MD, Mark C. Walters, MD, Matthew Hsieh, MD, Lakshmanan Krishnamurti, MD, Janet L. Kwiatkowski, MD, Rammurti Kamble, MD, Christof von Kalle, MD, Marcelyne Joseney-Antoine, Francis J. Pierciey, Jr., Weiliang Shi, PhD, Mohammed Asmal, MD PhD, Alexis A Thompson, MD MPH, John F Tisdale, MD

Blood (2017) 130 (Supplement 1): 527.

https://doi.org/10.1182/blood.V130.Suppl_1.527.527

Published: 2017

Journal Articles

Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia: Update from the Northstar Hgb-204 Phase 1/2 Clinical Study

Alexis A. Thompson, MD MPH, Janet Kwiatkowski, MD, John Rasko, MBBS, PhD, Suradej Hongeng, MD, Gary J. Schiller, MD, Usanarat Anurathapan, MD, Marina Cavazzana, MD PhD, P. Joy Ho, Christof von Kalle, MD, Morris Kletzel, MD FAAP, MBA, Philippe Leboulch, MD, Elliott Vichinsky, MD, Alexandria Petrusich, Mohammed Asmal, MD PhD, Mark C. Walters, MD

Blood (2016) 128 (22): 1175.

https://doi.org/10.1182/blood.V128.22.1175.1175

Published: 2016

Journal Articles

Interim Results from a Phase 1/2 Clinical Study of Lentiglobin Gene Therapy for Severe Sickle Cell Disease

Julie Kanter, MD, Mark C. Walters, MD, Matthew M. Hsieh, MD, Lakshmanan Krishnamurti, MD, Janet Kwiatkowski, MD, Rammurti T Kamble, MD, Christof von Kalle, MD, Frans A. Kuypers, PhD, Marina Cavazzana, MD PhD, Philippe Leboulch, MD, Marcelyne Joseney-Antoine, Mohammed Asmal, MD PhD, Alexis A. Thompson, MD MPH, John F. Tisdale, MD

Blood (2016) 128 (22): 1176.

https://doi.org/10.1182/blood.V128.22.1176.1176

Published: 2016

Journal Articles

Megatal, Crispr/Cas9, and Talen T-Cell Receptor Gene Editing

Mark J. Osborn, PhD, Beau R Webber, PhD, Friederike Knipping, Cara-lin Lonetree, Anthony DeFeo, Amber McElroy, Colby Starker, PhD, Richard Gabriel, Sarah Merkel, Keli L. Hippen, PhD, Daniel Voytas, PhD, Christof von Kalle, MD, Manfred Schmidt, PhD, Jeffrey S. Miller, MD, Jordan Jarjour, PhD, Alexander Astrakhan, PhD, Andrew M Scharenberg, MD, Jakub Tolar, MD, PhD, Bruce R. Blazar, MD

Blood (2015) 126 (23): 2045.

https://doi.org/10.1182/blood.V126.23.2045.2045

Published: 2015

Journal Articles

Update of Results from the Northstar Study (HGB-204): A Phase 1/2 Study of Gene Therapy for Beta-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex-Vivo with a Lentiviral Beta AT87Q-Globin Vector (LentiGlobin BB305 Drug Product)

Mark C. Walters, MD, John Rasko, MBBS, PhD, Suradej Hongeng, MD, Janet Kwiatkowski, MD, Gary J Schiller, MD, Morris Kletzel, MD, P. Joy Ho, MBBS, DPhil, Elliott Vichinsky, MD, Christof von Kalle, MD, Marina Cavazzana, MD PhD, Philippe Leboulch, MD, Alexandria Petrusich, Sandeep Soni, MD, Alexis A. Thompson, MD

Blood (2015) 126 (23): 201.

https://doi.org/10.1182/blood.V126.23.201.201

Published: 2015

Journal Articles

Outcomes of Gene Therapy for Severe Sickle Disease and Beta-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral Beta AT87Q-Globin Vector

Blood (2015) 126 (23): 202.

https://doi.org/10.1182/blood.V126.23.202.202

Published: 2015

Journal Articles

Initial Results from Study Hgb-206: A Phase 1 Study Evaluating Gene Therapy By Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with the Lentiglobin BB305 Lentiviral Vector in Subjects with Severe Sickle Cell Disease

Julie Kanter, MD, Mark C. Walters, MD, Matthew Hsieh, MD, Alexis A. Thompson, MD, Lakshmanan Krishnamurti, MD, Janet Kwiatkowski, MD, Rammurti T Kamble, MD, Christof von Kalle, MD, Frans A. Kuypers, PhD, Marina Cavazzana, MD PhD, Philippe Leboulch, MD, Laura Sandler, MPH, Sandeep Soni, MD, John F. Tisdale, MD

Blood (2015) 126 (23): 3233.

https://doi.org/10.1182/blood.V126.23.3233.3233

Published: 2015

Journal Articles

Tracking Genetically Engineered Lymphocytes Long-Term Reveals the Dynamics of T-Cell Immunological Memory

Blood (2015) 126 (23): 263.

https://doi.org/10.1182/blood.V126.23.263.263

Published: 2015

Journal Articles

Long-Term Immunological Profile of Patients Treated with Haploidentical HSCT and TK-Cells to Study the Requirements of Memory T Cell Persistence

Giacomo Oliveira, MSc, Maria Teresa Lupo Stanghellini, MD, Eliana Ruggiero, PhD, Nicoletta Cieri, MD PhD, Mattia D'Agostino, MD, Raffaella Greco, MD, Sara Mastaglio, MD, Maddalena Noviello, PhD, Luca Vago, MD PhD, Luca Biasco, PhD, Attilio Bondanza, MD PhD, Alessandro Aiuti, MD, Antonio Lambiase, MD, Manfred Schmidt, PhD, Catia Traversari, PhD, Christof von Kalle, MD, Fabio Ciceri, MD, Claudio Bordignon, MD, Chiara Bonini, MD

Blood (2014) 124 (21): 4793.

https://doi.org/10.1182/blood.V124.21.4793.4793

Published: 2014

Journal Articles

Study Hgb-205: Outcomes of Gene Therapy for Hemoglobinopathies Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral β Α-T87Q -Globin Vector (LentiGlobin ® BB305 Drug Product)

Blood (2014) 124 (21): 4797.

https://doi.org/10.1182/blood.V124.21.4797.4797

Published: 2014

Journal Articles

Initial Results from the Northstar Study (HGB-204): A Phase 1/2 Study of Gene Therapy for β-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral β Α-T87Q -Globin Vector (LentiGlobin BB305 Drug Product)

Alexis A. Thompson, MD, John E Rasko, MBBS, PhD, Suradej Hongeng, MD, Janet L. Kwiatkowski, MD MSCE, Gary Schiller, MD, Christof von Kalle, MD, Marina Cavazzana, MD PhD, Philippe Leboulch, MD, Alexandria Petrusich, Sandeep Soni, MD, Mark C. Walters, MD

Blood (2014) 124 (21): 549.

https://doi.org/10.1182/blood.V124.21.549.549

Published: 2014

Journal Articles

Hematopoietic Stem Cell Gene Therapy For Wiskott- Aldrich Syndrome

Blood (2013) 122 (21): 718.

https://doi.org/10.1182/blood.V122.21.718.718

Published: 2013

Journal Articles

Long-Term Immunological Profile and T Cell Dynamics In Patients Treated With Allogeneic Transplantation and TK-Cells For Hematological Malignancies

Giacomo Oliveira, MSc, Maria Teresa Lupo Stanghellini, MD, Nicoletta Cieri, MD, Raffaella Greco, MD, Maddalena Noviello, PhD, Veronica Valtolina, BSc, Luca Vago, MD, PhD, Catia Traversari, MD, Eliana Ruggiero, PhD, Anna Paruzynski, PhD, Manfred Schmidt, PhD, Christof von Kalle, MD, PhD, Fabio Ciceri, MD, Claudio Bordignon, MD, Chiara Bonini, MD

Blood (2013) 122 (21): 165.

https://doi.org/10.1182/blood.V122.21.165.165

Published: 2013

Journal Articles

Continued Response off Treatment After BRAF Inhibition in Refractory Hairy Cell Leukemia

Sascha Dietrich, MD, Jennifer Hüllein, Michael Hundemer, Nicola Lehners, MD, Alexander Jethwa, David Capper, MD, Till Acker, MD, Boyan Garvalov, PhD, Mindaugas Andrulis, MD, Carolin Blume, Julia Meissner, MD, Stefan Fröhling, MD, Christof von Kalle, MD, Hanno Glimm, MD, Anthony D. Ho, MD, Thorsten Zenz, MD

Blood (2012) 120 (21): 4600.

https://doi.org/10.1182/blood.V120.21.4600.4600

Published: 2012

Journal Articles

Overexpression of EVI1 Causes Genomic Instability and Cell Cycle Arrest in Hematopoietic Cells.

Friederike Herbst, PhD, Claudia R Ball, PhD, Oksana Zavidij, PhD, Annika Mengering, Sylvia Fessler, Manfred Schmidt, PhD, Christof von Kalle, MD, PhD, Hanno Glimm, MD

Blood (2012) 120 (21): 2398.

https://doi.org/10.1182/blood.V120.21.2398.2398

Published: 2012

Journal Articles

Deregulated EVI1 Expression Leads to Genomic Instability and G1 Cell Cycle Arrest

Friederike Herbst, Claudia R Ball, PhD, Wei Wang, Kadin Karakaya, Sylvia Fessler, Alwin Krämer, MD, Manfred Schmidt, PhD, Christof von Kalle, MD, Hanno Glimm, MD

Blood (2011) 118 (21): 2431.

https://doi.org/10.1182/blood.V118.21.2431.2431

Published: 2011

Journal Articles

Efficacy of Gene Therapy for Wiskott-Aldrich-Syndrome

Blood (2011) 118 (21): 165.

https://doi.org/10.1182/blood.V118.21.165.165

Published: 2011

Journal Articles

Impaired Lentiviral Transgene Expression In Vivo Caused by Massive Methylation of SFFV Promoter Sequences.

Friederike Herbst, Claudia R Ball, PhD, Francesca Tuorto, PhD, Wei Wang, Ulrich Kloz, Frank van der Hoeven, Frank Lyko, PhD, Christof Von Kalle, MD, Hanno Glimm, MD

Blood (2010) 116 (21): 3760.

https://doi.org/10.1182/blood.V116.21.3760.3760

Published: 2010

Journal Articles

In Vivo Lentiviral Marking Demonstrates Long-Term Myeloid and Lymphoid Lineage Contribution of Individual Hematopoietic Stem Cell Clones to Murine Steady-State Hematopoiesis.

Oksana Zavidij, Claudia R Ball, Sylvia Fessler, Daniela Belle, Manfred Schmidt, Christof von Kalle, MD, Hanno Glimm, MD

Blood (2009) 114 (22): 813.

https://doi.org/10.1182/blood.V114.22.813.813

Published: 2009

NARROW

Clinical Outcomes up to 3 Years Following Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar Hgb-204 Study

Interim Results from a Phase 1/2 Clinical Study of Lentiglobin Gene Therapy for Severe Sickle Cell Disease

Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia: Update from the Northstar Hgb-204 Phase 1/2 Clinical Study

Interim Results from a Phase 1/2 Clinical Study of Lentiglobin Gene Therapy for Severe Sickle Cell Disease

Megatal, Crispr/Cas9, and Talen T-Cell Receptor Gene Editing

Update of Results from the Northstar Study (HGB-204): A Phase 1/2 Study of Gene Therapy for Beta-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex-Vivo with a Lentiviral Beta AT87Q-Globin Vector (LentiGlobin BB305 Drug Product)

Outcomes of Gene Therapy for Severe Sickle Disease and Beta-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral Beta AT87Q-Globin Vector

Initial Results from Study Hgb-206: A Phase 1 Study Evaluating Gene Therapy By Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with the Lentiglobin BB305 Lentiviral Vector in Subjects with Severe Sickle Cell Disease

Tracking Genetically Engineered Lymphocytes Long-Term Reveals the Dynamics of T-Cell Immunological Memory

Long-Term Immunological Profile of Patients Treated with Haploidentical HSCT and TK-Cells to Study the Requirements of Memory T Cell Persistence

Study Hgb-205: Outcomes of Gene Therapy for Hemoglobinopathies Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral β Α-T87Q -Globin Vector (LentiGlobin ® BB305 Drug Product)

Initial Results from the Northstar Study (HGB-204): A Phase 1/2 Study of Gene Therapy for β-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral β Α-T87Q -Globin Vector (LentiGlobin BB305 Drug Product)

Hematopoietic Stem Cell Gene Therapy For Wiskott- Aldrich Syndrome

Long-Term Immunological Profile and T Cell Dynamics In Patients Treated With Allogeneic Transplantation and TK-Cells For Hematological Malignancies

Continued Response off Treatment After BRAF Inhibition in Refractory Hairy Cell Leukemia

Overexpression of EVI1 Causes Genomic Instability and Cell Cycle Arrest in Hematopoietic Cells.

Deregulated EVI1 Expression Leads to Genomic Instability and G1 Cell Cycle Arrest

Efficacy of Gene Therapy for Wiskott-Aldrich-Syndrome

Impaired Lentiviral Transgene Expression In Vivo Caused by Massive Methylation of SFFV Promoter Sequences.

In Vivo Lentiviral Marking Demonstrates Long-Term Myeloid and Lymphoid Lineage Contribution of Individual Hematopoietic Stem Cell Clones to Murine Steady-State Hematopoiesis.

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American Society of Hematology

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American Society of Hematology

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