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Journal Articles

Polyclonality Strongly Correlates with Biological Outcomes and Is Significantly Increased Following Improvements to the Phase 1/2 HGB-206 Protocol and Manufacturing of LentiGlobin for Sickle Cell Disease (SCD; bb1111) Gene Therapy (GT)

Blood (2021) 138 (Supplement 1): 561.

https://doi.org/10.1182/blood-2021-147760

Published: 2021

Journal Articles

Resolution of Serious Vaso-Occlusive Pain Crises and Reduction in Patient-Reported Pain Intensity: Results from the Ongoing Phase 1/2 HGB-206 Group C Study of LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy

Alexis A. Thompson, MD, Mark C. Walters, MD, Markus Y Mapara, MD PhD, Janet L. Kwiatkowski, MD MSCE, Lakshmanan Krishnamurti, MD, Banu Aygun, MD, Kimberly A. Kasow, DO, Stacey Rifkin-Zenenberg, DO, Manfred Schmidt, PhD, Jay DelCarpini, Francis J. Pierciey, Jr., Alexandra L. Miller, Meghan E. Gallagher, MSc, Ren Chen, Sunita Goyal, MD, Julie Kanter, MD, John F. Tisdale, MD

Blood (2020) 136 (Supplement 1): 16–17.

https://doi.org/10.1182/blood-2020-134940

Published: 2020

Journal Articles

Exploring the Drivers of Potential Clinical Benefit in Initial Patients Treated in the Hgb-206 Study of Lentiglobin for Sickle Cell Disease (SCD) Gene Therapy

Mark C. Walters, MD, John F. Tisdale, MD, Janet L. Kwiatkowski, MD MSCE, Lakshmanan Krishnamurti, MD, Markus Y. Mapara, MD PhD, Manfred Schmidt, PhD, Alexandra L. Miller, Francis J. Pierciey, Jr., Wenmei Huang, PhD MS, Jean-Antoine Ribeil, MD PhD, Julie Kanter, MD, Alexis A. Thompson, MD MPH

Blood (2019) 134 (Supplement_1): 2061.

https://doi.org/10.1182/blood-2019-128814

Published: 2019

Journal Articles

Hematopoietic Engraftment of Fanconi Anemia Patients through 3 Years after Gene Therapy

Blood (2019) 134 (Supplement_1): 4627.

https://doi.org/10.1182/blood-2019-129686

Published: 2019

Journal Articles

Long-Term Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar (HGB-204) Study

Janet L. Kwiatkowski, MD MSCE, Alexis A. Thompson, MD MPH, John E.J. Rasko, MBBS, PhD, Suradej Hongeng, MD, Gary J. Schiller, MD, Usanarat Anurathapan, MD, Marina Cavazzana, MD PhD, P. Joy Ho, MBBS DPhil FRACP FRCPA, Manfred Schmidt, PhD, Morris Kletzel, MD MBA, Elliott P. Vichinsky, MD, Briana Deary, Ying Chen, PhD, Alexandria Petrusich, Mark C. Walters, MD

Blood (2019) 134 (Supplement_1): 4628.

https://doi.org/10.1182/blood-2019-125807

Published: 2019

Journal Articles

Resolution of Sickle Cell Disease Manifestations in Patients Treated with Lentiglobin Gene Therapy: Updated Results from the Phase 1/2 Hgb-206 Group C Study

Julie Kanter, MD, John F. Tisdale, MD, Markus Y. Mapara, MD PhD, Janet L. Kwiatkowski, MD MSCE, Lakshmanan Krishnamurti, MD, Manfred Schmidt, PhD, Alexandra L. Miller, Francis J. Pierciey, Jr., Wenmei Huang, PhD MS, Jean-Antoine Ribeil, MD PhD, Alexis A. Thompson, MD MPH, Mark C. Walters, MD

Blood (2019) 134 (Supplement_1): 990.

https://doi.org/10.1182/blood-2019-128894

Published: 2019

Journal Articles

Advances in the Gene Therapy of Patients with Fanconi Anemia

Blood (2018) 132 (Supplement 1): 1022.

https://doi.org/10.1182/blood-2018-99-111160

Published: 2018

Journal Articles

Current Results of Lentiglobin Gene Therapy in Patients with Severe Sickle Cell Disease Treated Under a Refined Protocol in the Phase 1 Hgb-206 Study

John F. Tisdale, MD, Julie Kanter, MD, Markus Y. Mapara, MD PhD, Janet L. Kwiatkowski, MD, Lakshmanan Krishnamurti, MD, Manfred Schmidt, PhD, Alexandra L. Miller, Francis J. Pierciey, Jr., Weiliang Shi, PhD, Jean-Antoine Ribeil, MD PhD, Mohammed Asmal, MD PhD, Alexis A. Thompson, MD MPH, Mark C. Walters, MD

Blood (2018) 132 (Supplement 1): 1026.

https://doi.org/10.1182/blood-2018-99-113480

Published: 2018

Journal Articles

Outcomes for Initial Patient Cohorts with up to 33 Months of Follow-up in the Hgb-206 Phase 1 Trial

Julie Kanter, MD, John F. Tisdale, MD, Janet L. Kwiatkowski, MD, Lakshmanan Krishnamurti, MD, Markus Y. Mapara, MD PhD, Manfred Schmidt, PhD, Alexandra L. Miller, Francis J. Pierciey, Jr., Weiliang Shi, PhD, Jean-Antoine Ribeil, MD PhD, Mark C. Walters, MD, Alexis A. Thompson, MD MPH

Blood (2018) 132 (Supplement 1): 1080.

https://doi.org/10.1182/blood-2018-99-113477

Published: 2018

Journal Articles

Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia Following Completion of the Northstar HGB-204 Study

John E.J. Rasko, MBBS, PhD, Alexis A. Thompson, MD MPH, Janet L. Kwiatkowski, MD, Suradej Hongeng, MD, Gary J. Schiller, MD, Usanarat Anurathapan, MD, Marina Cavazzana, MD PhD, P. Joy Ho, MBBS DPhil FRACP FRCPA, Manfred Schmidt, PhD, Morris Kletzel, MD MBA, Philippe Leboulch, MD, Elliott Vichinsky, MD, Briana Deary, Ying Chen, PhD, Mohammed Asmal, MD PhD, Mark C. Walters, MD

Blood (2018) 132 (Supplement 1): 167.

https://doi.org/10.1182/blood-2018-167

Published: 2018

Journal Articles

IL-10 Receptor Deficiency Aggravates Exhaustion of CD8 + T-Cells and Impedes Their Control of Chronic Lymphocytic Leukemia

Bola S Hanna, PhD, Philipp M Röβner, Murat Iskar, PhD, Norman Mack, Verena Kalter, Manfred Schmidt, PhD, Marc Zapatka, PhD, Markus Feuerer, MD, Richard Gabriel, PhD, Peter Lichter, PhD, Martina Seiffert

Blood (2017) 130 (Supplement 1): 385.

https://doi.org/10.1182/blood.V130.Suppl_1.385.385

Published: 2017

Journal Articles

Identification and Validation of Novel Hematopoietic Stem Cell Regulatory microRNAs

Elias Simon Peter Eckert, Peer Wünsche, Manfred Schmidt, PhD, Claudia Ball, PhD, Friederike Herbst, PhD, Hanno Glimm

Blood (2016) 128 (22): 3872.

https://doi.org/10.1182/blood.V128.22.3872.3872

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

The Common Genomic Locus of Evl and MiR-342 Regulates Both Lymphopoiesis and Myelopoiesis

Tonio Lang, Friederike Herbst, PhD, Elias Eckert, Peer Wünsche, Shayda Hemmati, PhD, Tim Kindinger, Nina Hofmann, Oksana Zavidij, PhD, Manfred Schmidt, PhD, Claudia Ball, PhD, Hanno Glimm, MD

Blood (2015) 126 (23): 2366.

https://doi.org/10.1182/blood.V126.23.2366.2366

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

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

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

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

NARROW

Polyclonality Strongly Correlates with Biological Outcomes and Is Significantly Increased Following Improvements to the Phase 1/2 HGB-206 Protocol and Manufacturing of LentiGlobin for Sickle Cell Disease (SCD; bb1111) Gene Therapy (GT)

Resolution of Serious Vaso-Occlusive Pain Crises and Reduction in Patient-Reported Pain Intensity: Results from the Ongoing Phase 1/2 HGB-206 Group C Study of LentiGlobin for Sickle Cell Disease (bb1111) Gene Therapy

Exploring the Drivers of Potential Clinical Benefit in Initial Patients Treated in the Hgb-206 Study of Lentiglobin for Sickle Cell Disease (SCD) Gene Therapy

Hematopoietic Engraftment of Fanconi Anemia Patients through 3 Years after Gene Therapy

Long-Term Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar (HGB-204) Study

Resolution of Sickle Cell Disease Manifestations in Patients Treated with Lentiglobin Gene Therapy: Updated Results from the Phase 1/2 Hgb-206 Group C Study

Advances in the Gene Therapy of Patients with Fanconi Anemia

Current Results of Lentiglobin Gene Therapy in Patients with Severe Sickle Cell Disease Treated Under a Refined Protocol in the Phase 1 Hgb-206 Study

Outcomes for Initial Patient Cohorts with up to 33 Months of Follow-up in the Hgb-206 Phase 1 Trial

Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia Following Completion of the Northstar HGB-204 Study

IL-10 Receptor Deficiency Aggravates Exhaustion of CD8 + T-Cells and Impedes Their Control of Chronic Lymphocytic Leukemia

Identification and Validation of Novel Hematopoietic Stem Cell Regulatory microRNAs

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

The Common Genomic Locus of Evl and MiR-342 Regulates Both Lymphopoiesis and Myelopoiesis

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

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)

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

Hematopoietic Stem Cell Gene Therapy For Wiskott- Aldrich Syndrome

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American Society of Hematology

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American Society of Hematology

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