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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

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

Longer Term Follow-up on the First Patients with Severe Hemoglobinopathies Treated with Lentiglobin Gene Therapy

Blood (2017) 130 (Supplement 1): 4609.

https://doi.org/10.1182/blood.V130.Suppl_1.4609.4609

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

Update from the Hgb-205 Phase 1/2 Clinical Study of Lentiglobin Gene Therapy: Sustained Clinical Benefit in Severe Hemoglobinopathies

Blood (2016) 128 (22): 2311.

https://doi.org/10.1182/blood.V128.22.2311.2311

Published: 2016

Journal Articles

High-Efficiency Transduction of Primary Human Hematopoietic Stem/Progenitor Cells By AAV6 Vectors: strategies for Overcoming Donor-Variation and Implications in Genome Editing

Chen Ling, PhD, Kanit Bhukhai, PhD, Zifei Yin, MD PhD, Mengqun Tan, MD, Mervin Yoder, MD, Philippe Leboulch, MD, Emmanuel Payen, PhD, Arun Srivastava, PhD

Blood (2016) 128 (22): 5889.

https://doi.org/10.1182/blood.V128.22.5889.5889

Published: 2016

Journal Articles

Bench to Bedside-Gene Therapy for Thalassemia and Sickle Cell Disease

Philippe Leboulch, MD

Blood (2015) 126 (23): SCI-18.

https://doi.org/10.1182/blood.V126.23.SCI-18.SCI-18

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

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

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

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

Erosion Of The Chronic Myeloid Leukemia Stem Cell Pool By PPARγ Agonists

Stephane Prost, Francis Relouzat, Marc Spenchian, Gérald Massonnet, Jean-Paul Beressi, Els Verhoeyen, Benjamin Maneglier, Sylvie Castaigne, Christine Chomienne, Stany Chretien, Philippe Rousselot, Philippe Leboulch, PhD

Blood (2013) 122 (21): 5197.

https://doi.org/10.1182/blood.V122.21.5197.5197

Published: 2013

Journal Articles

Heat Shock Protein 70 Cytosolic Sequestration by Excess of Free Alpha-Globin Chains Is a Key Mechanism of the Ineffective Erythropoiesis in β-Thalassemia Major Patients

Jean-Benoît Arlet, MD, Jean-Antoine Ribeil, MD, PhD, Adonis Hazoumé, PhD, Samuel Demarest, Luba Tchertanov, PhD, Dussiot Michael, Ivan Cruz Moura, PhD, Thiago Maciel, PhD, Christian Auclair, PhD, Olivier Negre, PhD, Philippe Leboulch, MD, Véronique Baudin-creuza, PhD, Carmen Garrido, PhD, Genevieve Courtois, PhD, Olivier Hermine, MD, PhD

Blood (2012) 120 (21): 823.

https://doi.org/10.1182/blood.V120.21.823.823

Published: 2012

Journal Articles

HSC Transplantation for Hemoglobinopathies: Allogeneic or Autologous Gene-Modified HSC?

Marina Cavazzana-Calvo, MD, PhD, Emmanuel Payen, Olivier Negre, PhD, Salima Hacein-Bey-Abina, PharmD, PhD, Philippe Leboulch, PhD

Blood (2011) 118 (21): SCI-46.

https://doi.org/10.1182/blood.V118.21.SCI-46.SCI-46

Published: 2011

Journal Articles

Correction of murine β-thalassemia after minimal lentiviral gene transfer and homeostatic in vivo erythroid expansion

Olivier Negre, Floriane Fusil, Charlotte Colomb, Shoshannah Roth, Beatrix Gillet-Legrand, Annie Henri, Yves Beuzard, Frederic Bushman, Philippe Leboulch, Emmanuel Payen

Blood (2011) 117 (20): 5321–5331.

https://doi.org/10.1182/blood-2010-01-263582

Published: 2011

Includes: Supplemental data

Journal Articles

Is normal hematopoiesis maintained solely by long-term multipotent stem cells?

Marina Cavazzana-Calvo, Alain Fischer, Frederic D. Bushman, Emmanuel Payen, Salima Hacein-Bey-Abina, Philippe Leboulch

Blood (2011) 117 (17): 4420–4424.

https://doi.org/10.1182/blood-2010-09-255679

Published: 2011

Journal Articles

Dynamics of gene-modified progenitor cells analyzed by tracking retroviral integration sites in a human SCID-X1 gene therapy trial

Gary P. Wang, Charles C. Berry, Nirav Malani, Philippe Leboulch, Alain Fischer, Salima Hacein-Bey-Abina, Marina Cavazzana-Calvo, Frederic D. Bushman

Blood (2010) 115 (22): 4356–4366.

https://doi.org/10.1182/blood-2009-12-257352

Published: 2010

Includes: Supplemental data

Journal Articles

Thrombopoietin Administration Alleviates Hematopoietic Stem Cells Intrinsic Long Term Ionizing Radiation Damages. Identification of Preferential Cellular Expansion Sites

Diana Tronik-Le Roux, Johnny Nehme, Arthur Simonnet, Pierre Vaigot, Marie Anne Nicola, Paquita Nurden, Philippe Leboulch

Blood (2008) 112 (11): 2441.

https://doi.org/10.1182/blood.V112.11.2441.2441

Published: 2008

NARROW

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

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

Longer Term Follow-up on the First Patients with Severe Hemoglobinopathies Treated with Lentiglobin Gene Therapy

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

Update from the Hgb-205 Phase 1/2 Clinical Study of Lentiglobin Gene Therapy: Sustained Clinical Benefit in Severe Hemoglobinopathies

High-Efficiency Transduction of Primary Human Hematopoietic Stem/Progenitor Cells By AAV6 Vectors: strategies for Overcoming Donor-Variation and Implications in Genome Editing

Bench to Bedside-Gene Therapy for Thalassemia and Sickle Cell Disease

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

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

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)

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)

Erosion Of The Chronic Myeloid Leukemia Stem Cell Pool By PPARγ Agonists

Heat Shock Protein 70 Cytosolic Sequestration by Excess of Free Alpha-Globin Chains Is a Key Mechanism of the Ineffective Erythropoiesis in β-Thalassemia Major Patients

HSC Transplantation for Hemoglobinopathies: Allogeneic or Autologous Gene-Modified HSC?

Correction of murine β-thalassemia after minimal lentiviral gene transfer and homeostatic in vivo erythroid expansion

Is normal hematopoiesis maintained solely by long-term multipotent stem cells?

Dynamics of gene-modified progenitor cells analyzed by tracking retroviral integration sites in a human SCID-X1 gene therapy trial

Thrombopoietin Administration Alleviates Hematopoietic Stem Cells Intrinsic Long Term Ionizing Radiation Damages. Identification of Preferential Cellular Expansion Sites

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American Society of Hematology

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American Society of Hematology

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