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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

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

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

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

Modulation of Activin Signaling by RAP-011 (ActRIIA-IgG1) Improve Anemia, Increases Hemoglobin Levels and Corrects Ineffective Erythropoiesis in β-Thalassemia

Michael Dussiot, Thiago Maciel, PhD, Aurelie Fricot, Joel Veiga, Etienne Paubelle, Emmanuel Payen, Yves Beuzard, MD, Jean-Antoine Ribeil, MD, PhD, Jean-Benoît Arlet, MD, Genevieve Courtois, PhD, Tom O Daniel, MD, PhD, Rajesh Chopra, MD, PhD, Victoria Sung, Ph.D., Olivier Hermine, MD, PhD, Ivan Cruz Moura, PhD

Blood (2012) 120 (21): 247.

https://doi.org/10.1182/blood.V120.21.247.247

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

PDE-4 Inhibitor Rolipram Prevents Hypoxia Induced Pulmonary Hypertension in Transgenic Sickle Cell Sad Mice.

Lucia De Franceschi, MD, Giorgio Malpeli, Anne Janin, MD, Aldo Scarpa, MD, Christhophe Leboeuf, Orah Platt, MD, Philippe Leboulch, Emmanuel Payen, Yves Beuzard, MD, Carlo Brugnara, MD

Blood (2004) 104 (11): 3577.

https://doi.org/10.1182/blood.V104.11.3577.3577

Published: 2004

Journal Articles

Dimeric erythropoietin fusion protein with enhanced erythropoietic activity in vitro and in vivo

Bruno Dalle, Annie Henri, Philippe Rouyer-Fessard, Mickaël Bettan, Daniel Scherman, Yves Beuzard, Emmanuel Payen

Blood (2001) 97 (12): 3776–3782.

https://doi.org/10.1182/blood.V97.12.3776

Published: 2001

NARROW

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

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

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

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)

Modulation of Activin Signaling by RAP-011 (ActRIIA-IgG1) Improve Anemia, Increases Hemoglobin Levels and Corrects Ineffective Erythropoiesis in β-Thalassemia

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?

PDE-4 Inhibitor Rolipram Prevents Hypoxia Induced Pulmonary Hypertension in Transgenic Sickle Cell Sad Mice.

Dimeric erythropoietin fusion protein with enhanced erythropoietic activity in vitro and in vivo

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American Society of Hematology

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American Society of Hematology

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