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Journal Articles

SMART101 Donor T-Lymphoid Progenitors to Accelerate Immune Reconstitution Post-Haploidentical Peripheral Blood Stem Cell Transplantation with Post-Transplant Cyclophosphamide: SI101-02 First-in-Human Phase I/II

Raynier Devillier, Regis Peffault De Latour, Patrice Chevallier, Anne Huynh, Patrizia Chiusolo, Aurelie Bauquet, Laura Simons, Pierre Heimendinger, Olivier Negre, Shabi Soheili, Juliette Paillet, Isabelle Andre, Sebastien Oster, Marina Cavazzana, Frédéric François Lehmann, Fabio Ciceri

Blood (2023) 142 (Supplement 1): 4861.

https://doi.org/10.1182/blood-2023-179215

Published: 2023

Journal Articles

Human Pro-T-Cell Manufacturing in Vitro Is a Safe Procedure for Hematopoietic Stem Cell Transplantation with Delayed T-Cell Reconstitution: Interim Results from 2 Different Clinical Trials

Blood (2023) 142 (Supplement 1): 6868.

https://doi.org/10.1182/blood-2023-187607

Published: 2023

Journal Articles

Frequent Alterations of Driver Genes in Chromosome X and Their Clinical Relevance in Extranodal NK/T-Cell Lymphoma

Yuta Ito, Amira Marouf, Yasunori Kogure, Junji Koya, Mariko Tabata, Yuki Saito, Sumito Shingaki, Mitsuhiro Yuasa, Kentaro Yamaguchi, Julie Bruneau, Manon Vavasseur, Michaël Dussiot, Isabelle Andre, Akshay Joshi, Chantal Lagresle-Peyrou, Aude Magerus, Sammara Chaubard, David Lavergne, Emmanuel Bachy, Erika Brunet, Virginie Fataccioli, Chantal Brouzes, Camille Laurent, Laurence De Leval, Alexandra Traverse-Glehen, Céline Bossard, Marie Parrens, Véronique Meignin, Laure Philippe, Julien Rossignol, Felipe Suarez, Jean-Marie Michot, Olivier Tournilhac, Christine Bole-Feysot, Patrick Nitschke, Bruno Tesson, Cécile Laurent, Thierry Jo Molina, Vahid Asnafi, Sachiko Tsukita, Koji Izutsu, Hiroaki Miyoshi, Seiji Sakata, Akito Dobashi, Kengo Takeuchi, Koichi Ohshima, Philippe Gaulard, Arnaud Jaccard, Seishi Ogawa, Olivier Hermine, Keisuke Kataoka, Lucile Couronne

Blood (2022) 140 (Supplement 1): 1737–1739.

https://doi.org/10.1182/blood-2022-159869

Published: 2022

Journal Articles

An Autosomal Dominant Form of Ras-Related C3 Botulinum Toxin Substrate 2 (RAC2) Is Associated with Haematopoiesis Failure

Chantal Lagresle-Peyrou, Aurélien Olichon, Hanem Sadek, Philippe Roche, Claudine Tardy, Natael Sorel, Boris Bessot, Cindy Da Silva, Alain Fischer, Despina Moshous, Yves Collette, Capucine Picard, Jean-Laurent Casanova, Isabelle André, Marina Cavazzana

Blood (2021) 138 (Supplement 1): 4306.

https://doi.org/10.1182/blood-2021-150114

Published: 2021

Journal Articles

Rapid and Safe T Cell Immune Reconstitution By T Cell Progenitor Injection Following Haploidentical Transplantation for Severe Combined Immunodeficiency (SCID)

Despina Moshous, Elisa Magrin, Sarah Winter, Benjamin Fournier, Martin Castelle, Laurence Vendrame, Lucienne Chatenoud, Capucine Picard, Bénédicte Neven, Alessandra Magnani, Pierre Gaudeaux, Tayebeh-Shabi Soheili, Isabelle Andre, Laura Simons, Marina Cavazzana

Blood (2021) 138 (Supplement 1): 1752.

https://doi.org/10.1182/blood-2021-152281

Published: 2021

Journal Articles

Ex Vivo Production of Large Numbers of Genetically Modified NK Cells from Cord Blood or Mobilized Peripheral Blood CD34 + Cells Using Notch Ligand Delta-like 4 Culture System

Akshay Joshi, Isabelle Andre, Pierre Gaudeaux, Ranjita Devi Moirangthem, Tayebeh Shabi Soheili, Marina Cavazzana, Chantal Lagresle-Peyrou

Blood (2021) 138 (Supplement 1): 2778.

https://doi.org/10.1182/blood-2021-150293

Published: 2021

Journal Articles

A combination of cyclophosphamide and interleukin-2 allows CD4 + T cells converted to Tregs to control scurfy syndrome

Blood (2021) 137 (17): 2326–2336.

https://doi.org/10.1182/blood.2020009187

Published: 2021

Includes: Supplemental data

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

Editing a γ-Globin Repressor Binding Site Restores Fetal Hemoglobin Synthesis and Corrects the Phenotype of Sickle Cell Disease Erythrocytes

Leslie Weber, Giacomo Frati, Tristan Felix, Clara Wollenschlaeger, Antonio Casini, Vasco Meneghini, Cecile Masson, Anne De Cian, Anne Chalumeau, Fulvio Mavilio, PhD, Mario Amendola, Isabelle André, PhD, Anna Cereseto, Wassim El Nemer, PhD, Jean-Paul Concordet, Carine Giovannangeli, Marina Cavazzana, Annarita Miccio, PhD

Blood (2019) 134 (Supplement_1): 4635.

https://doi.org/10.1182/blood-2019-123192

Published: 2019

Journal Articles

An Autosomal Dominant SCID Form Due to a Gain of Function Mutation in the RAC2 Gene

Chantal Lagresle-Peyrou, PhD, Aurélien Olichon, Hanem Sadek, Philippe Roche, Claudine Tardy, Cindy Da Silva, Alexandrine Garrigue, Alain Fischer, MD PhD, Despina Moshous, MD PhD, Yves Collette, Capucine Picard, MD PhD, Jean-Laurent Casanova, PhD MD, Isabelle André, PhD, Marina Cavazzana, MD PhD

Blood (2019) 134 (Supplement_1): 3742.

https://doi.org/10.1182/blood-2019-126855

Published: 2019

Journal Articles

Modeling of Immune Reconstitution Post CD34 Selected Stem Cell Transplantation in Pediatric Patients with Severe Combined Immune Deficiency

Jean-Sebastien Diana, MD, Naim Bouazza, PhD, Chloé Couzin, MD, Martin Castelle, MD, Alessandra Magnani, MD PhD, Elisa Magrin, PhD, Laure Joseph, MD, Marianne Delville, MD, Francois Lefrere, MD, jean-Marc Treluyer, MD PhD, Capucine Picard, MD PhD, Despina Moshous, MD PhD, Isabelle André, PhD, Stephane Blanche, MD, Benedicte Neven, MD PhD, Marina Cavazzana, MD PhD

Blood (2019) 134 (Supplement_1): 5668.

https://doi.org/10.1182/blood-2019-127974

Published: 2019

Journal Articles

Analysis of RBC Properties in Patients with SCD Treated with Lentiglobin Gene Therapy

Nicolas Hebert, Elisa Magrin, PhD, Annarita Miccio, PhD, Kiger Laurent, PhD, Nguyen-Peyre Kim-Anh, PhD, Laure Joseph, MD, Alessandra Magnani, MD PhD, Chloé Couzin, MD, Wassim El Nemer, PhD, France Pirenne, MD PhD, Olivier Negre, PhD, Jean-Antoine Ribeil, MD PhD, Isabelle André-Schmutz, PhD, Pablo Bartolucci, MD PhD, Marina Cavazzana, MD PhD

Blood (2018) 132 (Supplement 1): 2195.

https://doi.org/10.1182/blood-2018-99-115973

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

Gene-corrected human Munc13-4–deficient CD8 + T cells can efficiently restrict EBV-driven lymphoproliferation in immunodeficient mice

Tayebeh Soheili, Julie Rivière, Ida Ricciardelli, Amandine Durand, Els Verhoeyen, Anne-Céline Derrien, Chantal Lagresle-Peyrou, Geneviève de Saint Basile, François-Loïc Cosset, Persis Amrolia, Isabelle André-Schmutz, Marina Cavazzana

Blood (2016) 128 (24): 2859–2862.

https://doi.org/10.1182/blood-2016-07-729871

Published: 2016

Includes: Supplemental data

Journal Articles

Generation of Functional Regulatory T Cells By FOXP3 Gene Transfer into CD4 T Cells from Scurfy Mice and IPEX Patients

Marianne Delville, MD, Emmanuelle Six, PhD, Florence Bellier, Nelly Sigrist, David Zemmour, PhD, Marie-André Bessard, MD, Fabienne Mazerolles, PhD, David Gross, PhD, Axel Schambach, MD PhD, Frederic Rieux-Laucat, PhD, Christoph Benoist, MD PhD, Isabelle Andre-Schmutz, Marina Cavazzana, MD PhD

Blood (2016) 128 (22): 2526.

https://doi.org/10.1182/blood.V128.22.2526.2526

Published: 2016

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

Treatment of Post- HSCT Immunodeficiency By Infusion of Ex Vivo- Generated T Cell Precursors from Adult and Cord Blood Hematopoietic Stem and Progenitor Cells

Laura Simons, MDPhD, Corinne De La Chappedelaine, Christian Reimann, MDPhD, Elodie Elkaim, MD, Sandrine Susini, PhD, Kuiying Ma, Fabien Touzot, MDPhD, Marina Cavazzana, MD PhD, Isabelle Andrè-Schmutz, PhD

Blood (2015) 126 (23): 1887.

https://doi.org/10.1182/blood.V126.23.1887.1887

Published: 2015

Journal Articles

From the Bone Marrow to the Thymic Niche

Sandrine Susini, Séverine Mouraud, Elodie Elkaim, Julien Roullier, Sonia Luce, Olivier Pellé, Julie Bruneau, Marina Cavazzana, MD PhD, Isabelle Andre-Schmutz, PhD

Blood (2014) 124 (21): 5123.

https://doi.org/10.1182/blood.V124.21.5123.5123

Published: 2014

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

Cytokine Environement Analysis During Allogeneic Hematopoietic Stem Cell Transplantation for Inherited Diseases

Fabien Touzot, MD, PhD, Bryan Fiema, Sophie Paczesny, M.D., Ph.D, Isabelle Andre-Schmutz, PhD, Dominique Helley, MD, PhD, Despina Moshous, MD, PhD, Corinne De Chappedelaine, Stephane Blanche, MD, Alain Fischer, MD/PhD, Marina Cavazzana-Calvo, MD, PhD

Blood (2012) 120 (21): 4484.

https://doi.org/10.1182/blood.V120.21.4484.4484

Published: 2012

NARROW

SMART101 Donor T-Lymphoid Progenitors to Accelerate Immune Reconstitution Post-Haploidentical Peripheral Blood Stem Cell Transplantation with Post-Transplant Cyclophosphamide: SI101-02 First-in-Human Phase I/II

Human Pro-T-Cell Manufacturing in Vitro Is a Safe Procedure for Hematopoietic Stem Cell Transplantation with Delayed T-Cell Reconstitution: Interim Results from 2 Different Clinical Trials

Frequent Alterations of Driver Genes in Chromosome X and Their Clinical Relevance in Extranodal NK/T-Cell Lymphoma

An Autosomal Dominant Form of Ras-Related C3 Botulinum Toxin Substrate 2 (RAC2) Is Associated with Haematopoiesis Failure

Rapid and Safe T Cell Immune Reconstitution By T Cell Progenitor Injection Following Haploidentical Transplantation for Severe Combined Immunodeficiency (SCID)

Ex Vivo Production of Large Numbers of Genetically Modified NK Cells from Cord Blood or Mobilized Peripheral Blood CD34 + Cells Using Notch Ligand Delta-like 4 Culture System

A combination of cyclophosphamide and interleukin-2 allows CD4 + T cells converted to Tregs to control scurfy syndrome

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

Editing a γ-Globin Repressor Binding Site Restores Fetal Hemoglobin Synthesis and Corrects the Phenotype of Sickle Cell Disease Erythrocytes

An Autosomal Dominant SCID Form Due to a Gain of Function Mutation in the RAC2 Gene

Modeling of Immune Reconstitution Post CD34 Selected Stem Cell Transplantation in Pediatric Patients with Severe Combined Immune Deficiency

Analysis of RBC Properties in Patients with SCD Treated with Lentiglobin Gene Therapy

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

Gene-corrected human Munc13-4–deficient CD8 + T cells can efficiently restrict EBV-driven lymphoproliferation in immunodeficient mice

Generation of Functional Regulatory T Cells By FOXP3 Gene Transfer into CD4 T Cells from Scurfy Mice and IPEX Patients

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

Treatment of Post- HSCT Immunodeficiency By Infusion of Ex Vivo- Generated T Cell Precursors from Adult and Cord Blood Hematopoietic Stem and Progenitor Cells

From the Bone Marrow to the Thymic Niche

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

Cytokine Environement Analysis During Allogeneic Hematopoietic Stem Cell Transplantation for Inherited Diseases

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American Society of Hematology

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