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Journal Articles

Preclinical Assessment for Gene Addition Strategy in Familial Hemophagocytic Lymphohistiocytosis Related to Munc 13-4 Deficiency

Chantal Lagresle-Peyrou, Jean-Sebastien Diana, Elisa Magrin, Clotilde Aussel, Natael Sorel, Yetki Aslan, Cécile Roudaut, Eden Schwartz, Aurélie Gabrion, Caroline Tuchmann-Durand, Francois Lefrere, Anne Galy, Despina Moshous, Marina Cavazzana

Blood (2023) 142 (Supplement 1): 3633.

https://doi.org/10.1182/blood-2023-186133

Published: 2023

Journal Articles

Outcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome

Blood (2023) 142 (15): 1281–1296.

https://doi.org/10.1182/blood.2022019117

Published: 2023

Includes: Supplemental data

Journal Articles

Long-Term Follow-up Study after Lentiviral Hematopoietic Stem/Progenitor Cell Gene Therapy for Wiskott - Aldrich Syndrome

Blood (2021) 138 (Supplement 1): 2934.

https://doi.org/10.1182/blood-2021-147697

Published: 2021

Journal Articles

Clonal tracking in gene therapy patients reveals a diversity of human hematopoietic differentiation programs

Blood (2020) 135 (15): 1219–1231.

https://doi.org/10.1182/blood.2019002350

Published: 2020

Includes: Supplemental data

Journal Articles

Outcome of Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome

Blood (2019) 134 (Supplement_1): 4629.

https://doi.org/10.1182/blood-2019-126161

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

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

Diverse and Distinct Human Hematopoietic Differentiation Programs Generate By Bone Marrow and G-CSF Mobilized Hematopoietic Stem and Progenitors Cells

Blood (2017) 130 (Supplement 1): 4618.

https://doi.org/10.1182/blood.V130.Suppl_1.4618.4618

Published: 2017

Journal Articles

Engraftment and in vivo proliferation advantage of gene-corrected mobilized CD34 + cells from Fanconi anemia patients

Paula Río, Susana Navarro, Guillermo Guenechea, Rebeca Sánchez-Domínguez, Maria Luisa Lamana, Rosa Yañez, Jose A. Casado, Parinda A. Mehta, Maria Roser Pujol, Jordi Surrallés, Sabine Charrier, Anne Galy, José C. Segovia, Cristina Díaz de Heredia, Julián Sevilla, Juan A. Bueren

Blood (2017) 130 (13): 1535–1542.

https://doi.org/10.1182/blood-2017-03-774174

Published: 2017

Includes: Supplemental data

Journal Articles

Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult

Blood (2017) 130 (11): 1327–1335.

https://doi.org/10.1182/blood-2017-04-777136

Published: 2017

Includes: Supplemental data

Journal Articles

a Diversity of Human Hematopoietic Differentiation Programs Identified through In Vivo Tracking of Hematopoiesis in Wiskott-Aldrich Syndrome Patients

Emmanuelle Six, PhD, Laure Caccavelli, Arnaud Lecoules, Christopher L Nobles, PhD, France Male, Cécile Roudaut, MSc, Clemence Plantier, Alessandra Magnani, MD PhD, Elisa Magrin, PhD, Nicolas Cagnard, Anne Galy, PharmD, PhD, Adrian J Thrasher, MD PhD, Salima hacein-Bey Abina, Agathe Guilloux, Isabelle Andre-Schmutz, Charles C. Berry, frederic D. Bushman, Marina Cavazzana, MD PhD

Blood (2016) 128 (22): 3871.

https://doi.org/10.1182/blood.V128.22.3871.3871

Published: 2016

Journal Articles

Gene Therapy Using a Self-Inactivating Lentiviral Vector Improves Clinical and Laboratory Manifestations of Wiskott-Aldrich Syndrome

Blood (2015) 126 (23): 260.

https://doi.org/10.1182/blood.V126.23.260.260

Published: 2015

Journal Articles

Stem Cell and T-Cell Gene Therapy Using SIN-Lentiviral Vector In Type 3 Familial Hemophagocytic Lymphohistiocytosis

Julie Rivière, Tayebeh Soheili, Els Verhoeyen, Anne Galy, Geneviève de Saint Basile, Alain Fischer, Isabelle Andre-Schmutz, Marina Cavazzana

Blood (2013) 122 (21): 4214.

https://doi.org/10.1182/blood.V122.21.4214.4214

Published: 2013

Journal Articles

Modeling JAK2 V617F Heterozygous and Homozygous Mutations by Using Induced Pluripotent Stem Cells Derived From Myeloproliferative Neoplasms Patients

Joseph Saliba, PhD Student, Sofiane Hamidi, PhD Student, Antonio Di Stefano, PhD, Thierry Langlois, Gaëlle Lenglet, PhD, Lise Secardin, Anne Galy, PharmD, PhD, Paule Opolon, Najet Debili, PhD, Hana Raslova, PhD, Françoise Norol, Isabelle Plo, Ph.D, William Vainchenker, MD, PhD

Blood (2012) 120 (21): 1758.

https://doi.org/10.1182/blood.V120.21.1758.1758

Published: 2012

Journal Articles

Abnormal Proplatelet Formation and Myosin Activation Status in Patients with MYH9-Related Syndrome

Yolande Chen, MD, Siham Boukour, PhD, Paquita Nurden, MD, PhD, Ababacar Seye, Olivier Bluteau, PhD, Alberta Palazzo, Yunhua Chang, PhD, Larissa Lordier, PhD, Remi Favier, MD, Nicole Schlegel, MD, PhD, Anne Galy, PharmD, PhD, Alan T. Nurden, PhD, Hana Raslova, PhD, William Vainchenker, MD, PhD, Najet Debili, PhD

Blood (2011) 118 (21): 4826.

https://doi.org/10.1182/blood.V118.21.4826.4826

Published: 2011

Journal Articles

A major role of TGF-β1 in the homing capacities of murine hematopoietic stem cell/progenitors

Claude Capron, Catherine Lacout, Yann Lécluse, Valérie Jalbert, Hédia Chagraoui, Sabine Charrier, Anne Galy, Annelise Bennaceur-Griscelli, Elisabeth Cramer-Bordé, William Vainchenker

Blood (2010) 116 (8): 1244–1253.

https://doi.org/10.1182/blood-2009-05-221093

Published: 2010

Includes: Supplemental data

Journal Articles

In Vitro Knock-Down of Myh9 Leads to a Myh9-Related Disorder Phenotype in Human Megakaryocytes.

Yolande Chen, MD, Siham Boukour, PhD, Monia Romdhane, Ababacar Seye, PhD, Olivier Bluteau, PhD, Larissa Lordier, PhD, Yunhua Chang, PhD, Anne Galy, D., Pharm, and, PhD, Rémi Favier, MD, Paquita Nurden, MD, Hana Raslova, PhD, William Vainchenker, MD, PhD, Najet Debili, PhD

Blood (2009) 114 (22): 4596.

https://doi.org/10.1182/blood.V114.22.4596.4596

Published: 2009

Journal Articles

Selective reduction of JAK2 V617F -dependent cell growth by siRNA/shRNA and its reversal by cytokines

Abire Jedidi, Caroline Marty, Charleen Oligo, Laurence Jeanson-Leh, Jean-Antoine Ribeil, Nicole Casadevall, Anne Galy, William Vainchenker, Jean-Luc Villeval

Blood (2009) 114 (9): 1842–1851.

https://doi.org/10.1182/blood-2008-09-176875

Published: 2009

Journal Articles

Constitutive TGF-β1 Deficiency Induces a Defect in Hematopoietic Stem/Progenitor Cell Compartment in Fetus and Neonate.

Claude Capron, Catherine Lacout, Yann Lecluse, Valérie Jalbert, Elisabeth Cramer Bordé, Anne Galy, Sabine Charrier, William Vainchenker

Blood (2008) 112 (11): 1397.

https://doi.org/10.1182/blood.V112.11.1397.1397

Published: 2008

Journal Articles

Evidence for Efficacy and Safety of Lentiviral Mediated Gene Transfer in T Cells and CD34+ Cells from Wiskott-Aldrich Syndrome Patients.

Samantha Scaramuzza, Sara Trifari, Francesco Marangoni, Silvana Martino, Ayse Metin, Anne Galy, Luigi Naldini, Alessandro Aiuti, Anna Villa, Loic Dupré, Maria Grazia Roncarolo

Blood (2006) 108 (11): 3279.

https://doi.org/10.1182/blood.V108.11.3279.3279

Published: 2006

NARROW

Preclinical Assessment for Gene Addition Strategy in Familial Hemophagocytic Lymphohistiocytosis Related to Munc 13-4 Deficiency

Outcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome

Long-Term Follow-up Study after Lentiviral Hematopoietic Stem/Progenitor Cell Gene Therapy for Wiskott - Aldrich Syndrome

Clonal tracking in gene therapy patients reveals a diversity of human hematopoietic differentiation programs

Outcome of Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome

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

Advances in the Gene Therapy of Patients with Fanconi Anemia

Diverse and Distinct Human Hematopoietic Differentiation Programs Generate By Bone Marrow and G-CSF Mobilized Hematopoietic Stem and Progenitors Cells

Engraftment and in vivo proliferation advantage of gene-corrected mobilized CD34 + cells from Fanconi anemia patients

Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult

a Diversity of Human Hematopoietic Differentiation Programs Identified through In Vivo Tracking of Hematopoiesis in Wiskott-Aldrich Syndrome Patients

Gene Therapy Using a Self-Inactivating Lentiviral Vector Improves Clinical and Laboratory Manifestations of Wiskott-Aldrich Syndrome

Stem Cell and T-Cell Gene Therapy Using SIN-Lentiviral Vector In Type 3 Familial Hemophagocytic Lymphohistiocytosis

Modeling JAK2 V617F Heterozygous and Homozygous Mutations by Using Induced Pluripotent Stem Cells Derived From Myeloproliferative Neoplasms Patients

Abnormal Proplatelet Formation and Myosin Activation Status in Patients with MYH9-Related Syndrome

A major role of TGF-β1 in the homing capacities of murine hematopoietic stem cell/progenitors

In Vitro Knock-Down of Myh9 Leads to a Myh9-Related Disorder Phenotype in Human Megakaryocytes.

Selective reduction of JAK2 V617F -dependent cell growth by siRNA/shRNA and its reversal by cytokines

Constitutive TGF-β1 Deficiency Induces a Defect in Hematopoietic Stem/Progenitor Cell Compartment in Fetus and Neonate.

Evidence for Efficacy and Safety of Lentiviral Mediated Gene Transfer in T Cells and CD34+ Cells from Wiskott-Aldrich Syndrome Patients.

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American Society of Hematology

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ASH Publications

American Society of Hematology

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