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Gene Therapy in a Patient with Sickle Cell Disease

The New England journal of medicine, 2017-03, Vol.376 (9), p.848-855

2017

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Sickle Hemoglobin Confers Tolerance to Plasmodium Infection

Cell, 2011-04, Vol.145 (3), p.398-409

2011

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Endothelin receptor antagonism prevents hypoxia-induced mortality and morbidity in a mouse model of sickle-cell disease

The Journal of clinical investigation, 2008-05, Vol.118 (5), p.1924-1933

2008

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Haemoglobin and haemoglobinopathies at the forefront in the 21st century

Hématologie, 2021-04, Vol.27 (2), p.55-77

Beuzard, Yves

2021

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Exquisite sensitivity of TP53 mutant and basal breast cancers to a dose-dense epirubicin-cyclophosphamide regimen

PLoS medicine, 2007-03, Vol.4 (3), p.e90

2007

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ICA-17043, a novel Gardos channel blocker, prevents sickled red blood cell dehydration in vitro and in vivo in SAD mice

Blood, 2003-03, Vol.101 (6), p.2412-2418

2003

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Permanent and Panerythroid Correction of Murine β Thalassemia by Multiple Lentiviral Integration in Hematopoietic Stem Cells

Proceedings of the National Academy of Sciences - PNAS, 2002-10, Vol.99 (22), p.14380-14385

2002

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Abnormal modulation of cell protective systems in response to ischemic/reperfusion injury is important in the development of mouse sickle cell hepatopathy

Haematologica (Roma), 2011-01, Vol.96 (1), p.24-32

2011

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Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia

The New England journal of medicine, 2018-04, Vol.378 (16), p.1479-1493

2018

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Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the β(A(T87Q))-Globin Gene

Human gene therapy, 2016-02, Vol.27 (2), p.148-165

2016

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An activin receptor IIA ligand trap corrects ineffective erythropoiesis in β-thalassemia

Nature medicine, 2014-04, Vol.20 (4), p.398-407

2014

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Sickle cell disease and risk of haematological malignancies

Hématologie, 2021-08, Vol.27 (4), p.173-187

2021

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Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial

Nature medicine, 2022-01, Vol.28 (1), p.81-88

2022

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Interest of a New Method for Free Plasma Heme Related Species Dosages in Sickle Cell Disease and Beta Thalassemia

Blood, 2019-11, Vol.134 (Supplement_1), p.3381-3381

2019

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HSP70 sequestration by free α-globin promotes ineffective erythropoiesis in β-thalassaemia

Nature (London), 2014-10, Vol.514 (7521), p.242-246

2014

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A gene therapy approach to regulated delivery of erythropoietin as a function of oxygen tension

Human gene therapy, 1997-11, Vol.8 (16), p.1881-1889

1997

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Stress-induced aberrant splicing of TSG101: association to high tumor grade and p53 status in breast cancers

Oncogene, 1999-12, Vol.18 (54), p.7834-7837

1999

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Dosage de l’hémolyse dans les anémies hémolytiques et le DHTR

Transfusion clinique et biologique : journal de la Société française de transfusion sanguine, 2021-11, Vol.28 (4), p.S105

2021

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Distribution of Lentiviral Vector Integration Sites in Mice Following Therapeutic Gene Transfer to Treat β-thalassemia

Molecular therapy, 2011-07, Vol.19 (7), p.1273-1286

2011

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Lentivirus Vectors in β-Thalassemia

Methods in Enzymology, 2012, Vol.507, p.109-124

2012

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Correction of Sickle Cell Disease in Transgenic Mouse Models by Gene Therapy

Science (American Association for the Advancement of Science), 2001-12, Vol.294 (5550), p.2368-2371

2001

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

Blood, 2011-05, Vol.117 (20), p.5321-5331

2011

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Gene Therapy in a Patient with Sickle Cell Disease

Sickle Hemoglobin Confers Tolerance to Plasmodium Infection

Preclinical evaluation of efficacy and safety of an improved lentiviral vector for the treatment of β-thalassemia and sickle cell disease

Endothelin receptor antagonism prevents hypoxia-induced mortality and morbidity in a mouse model of sickle-cell disease

Haemoglobin and haemoglobinopathies at the forefront in the 21st century

Exquisite sensitivity of TP53 mutant and basal breast cancers to a dose-dense epirubicin-cyclophosphamide regimen

ICA-17043, a novel Gardos channel blocker, prevents sickled red blood cell dehydration in vitro and in vivo in SAD mice

Permanent and Panerythroid Correction of Murine β Thalassemia by Multiple Lentiviral Integration in Hematopoietic Stem Cells

Abnormal modulation of cell protective systems in response to ischemic/reperfusion injury is important in the development of mouse sickle cell hepatopathy

Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia

Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the β(A(T87Q))-Globin Gene

An activin receptor IIA ligand trap corrects ineffective erythropoiesis in β-thalassemia

Sickle cell disease and risk of haematological malignancies

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial

Interest of a New Method for Free Plasma Heme Related Species Dosages in Sickle Cell Disease and Beta Thalassemia

HSP70 sequestration by free α-globin promotes ineffective erythropoiesis in β-thalassaemia

A gene therapy approach to regulated delivery of erythropoietin as a function of oxygen tension

Stress-induced aberrant splicing of TSG101: association to high tumor grade and p53 status in breast cancers

Dosage de l’hémolyse dans les anémies hémolytiques et le DHTR

Distribution of Lentiviral Vector Integration Sites in Mice Following Therapeutic Gene Transfer to Treat β-thalassemia

Lentivirus Vectors in β-Thalassemia

Correction of Sickle Cell Disease in Transgenic Mouse Models by Gene Therapy

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

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

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