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Journal Articles

Rac’n’rolling the immune system

Fabien Touzot

Blood (2024) 143 (15): 1433–1434.

https://doi.org/10.1182/blood.2023023630

Published: 2024

Journal Articles

Genotype, oxidase status, and preceding infection or autoinflammation do not affect allogeneic HCT outcomes for CGD

Jennifer W. Leiding, Danielle E. Arnold, Suhag Parikh, Brent Logan, Rebecca A. Marsh, Linda M. Griffith, Ruizhe Wu, Sharon Kidd, Kanwaldeep Mallhi, Deepak Chellapandian, Stephanie J. Si Lim, Eyal Grunebaum, E. Liana Falcone, Luis Murguia-Favela, Debbi Grossman, Vinod K. Prasad, Jennifer R. Heimall, Fabien Touzot, Lauri M. Burroughs, Jack Bleesing, Neena Kapoor, Jasmeen Dara, Olatundun Williams, Malika Kapadia, Benjamin R. Oshrine, Jeffrey J. Bednarski, Ahmad Rayes, Hey Chong, Geoffrey D. E. Cuvelier, Lisa R. Forbes Satter, Caridad Martinez, Mark T. Vander Lugt, Lolie C. Yu, Shanmuganathan Chandrakasan, Avni Joshi, Susan E. Prockop, Blachy J. Dávila Saldaña, Victor Aquino, Larisa A. Broglie, Christen L. Ebens, Lisa M. Madden, Kenneth DeSantes, Jordan Milner, Hemalatha G. Rangarajan, Ami J. Shah, Alfred P. Gillio, Alan P. Knutsen, Holly K. Miller, Theodore B. Moore, Pamela Graham, Andrea Bauchat, Nancy J. Bunin, Pierre Teira, Aleksandra Petrovic, Sharat Chandra, Hisham Abdel-Azim, Morna J. Dorsey, Olga Birbrayer, Morton J. Cowan, Christopher C. Dvorak, Elie Haddad, Donald B. Kohn, Luigi D. Notarangelo, Sung-Yun Pai, Jennifer M. Puck, Michael A. Pulsipher, Troy R. Torgerson, Harry L. Malech, Elizabeth M. Kang

Blood (2023) 142 (24): 2105–2118.

https://doi.org/10.1182/blood.2022019586

Published: 2023

Includes: Supplemental data

Journal Articles

Inherited human Apollo deficiency causes severe bone marrow failure and developmental defects

Blood (2022) 139 (16): 2427–2440.

https://doi.org/10.1182/blood.2021010791

Published: 2022

Includes: Supplemental data

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

Optimizing CD4+ T-Cells Transduction Protocol for Gene Therapy of HIV-1 Infected Patients

Amani Ouedrani, PharmD, Lounes Djerroudi, MD, Isabelle Hmitou, PhD, Marina Cavazzana, MD PhD, Fabien Touzot, MDPhD

Blood (2015) 126 (23): 4429.

https://doi.org/10.1182/blood.V126.23.4429.4429

Published: 2015

Journal Articles

Reiterated Therapeutic Drug Monitoring (TDM) Dosing to Significantly Improve the Control of Exposure to IV Busulfan in Infants and Older Children Undergoing Hematopoietic Stem-Cell Transplantation (HSCT)

Alexandre Amin, PharmD, Philippe Bourget, PharmD, PhD, Benedicte Neven, MD PhD, Marie-Louise Fremond, MD, Martin Castelle, MD, Elodie Elkaim, MD, Guilhem Cros, MD, Despina Moshous, MD, Fabien Touzot, MD, Alexandre Lalli, PharmD, Aurélie Petain, Laurent Nguyen, Stephane Blanche

Blood (2015) 126 (23): 4326.

https://doi.org/10.1182/blood.V126.23.4326.4326

Published: 2015

Journal Articles

B Cell Reconstitution after Gene Therapy in Patients with Wiskott Aldrich Syndrome and Comparison with Mismatched Allogeneic Hematopoietic Stem Cell Transplantation

Alessandra Magnani, MD PhD, Cécile Roudaut, MSc, Aurelie Gabrion, Laure Caccavelli, PhD, Fabien Touzot, MDPhD, Benedicte Neven, MD PhD, Despina Moshous, MD, Nizar Mahlaoui, Emmanuel Clave, Antoine Toubert, Alain Fischer, MD PhD, Marina Cavazzana, MD PhD

Blood (2015) 126 (23): 3235.

https://doi.org/10.1182/blood.V126.23.3235.3235

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

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

Faster T-cell development following gene therapy compared with haploidentical HSCT in the treatment of SCID-X1

Fabien Touzot, Despina Moshous, Rita Creidy, Bénédicte Neven, Pierre Frange, Guilhem Cros, Laure Caccavelli, Johanna Blondeau, Alessandra Magnani, Jean-Marc Luby, Brigitte Ternaux, Capucine Picard, Stéphane Blanche, Alain Fischer, Salima Hacein-Bey-Abina, Marina Cavazzana

Blood (2015) 125 (23): 3563–3569.

https://doi.org/10.1182/blood-2014-12-616003

Published: 2015

Includes: Supplemental data

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

Circulating Endothelial Cells As a Reliable Marker Of Endothelial Damage In Children Undergoing Hematopoietic Stem Cell Transplantation

Fabien Touzot, MD, PhD, Despina Moshous, MD, PhD, Guilhem Cros, MD, Pierre Frange, MD, Sebastien Heritier, MD, Benedicte Neven, Marina Cavazzana-Calvo, MD, PhD, Alain Fischer, MD/PhD, Stephane Blanche, MD, Dominique Helley, MD, PhD

Blood (2013) 122 (21): 2049.

https://doi.org/10.1182/blood.V122.21.2049.2049

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

Journal Articles

Anti CMV and/or Anti Adenovirus IFN-g-Positive CD4+ CD8+ T Lymphocytes for Treatment of Viral Infections After Allogeneic HSC Transplantation: First Results

Liliane Dal Cortivo, MD, RITA Creidy, PhD, Aurélie Gabrion, Marianne Leruez-Ville, Sebastien Heritier, MD, Fabien Touzot, MD, PhD, Guilhem Cros, MD, Sebastien Maury, MD, Alienor Xhaard, Marie Ouachee, MD, Ingrid Hamon, Tech, Capucine Picard, MD, PhD, Christophe Cambouris, Salima Hacein Bey Abina, PhD, Stephane Blanche, MD, Marina Cavazzana-Calvo, MD, PhD

Blood (2012) 120 (21): 1906.

https://doi.org/10.1182/blood.V120.21.1906.1906

Published: 2012

Journal Articles

Massive expansion of maternal T cells in response to EBV infection in a patient with SCID-Xl

Fabien Touzot, Liliane Dal-Cortivo, Virginie Verkarre, Annick Lim, Anne Crucis-Armengaud, Despina Moshous, Sébastien Héritier, Pierre Frange, Sophie Kaltenbach, Stéphane Blanche, Capucine Picard, Salima Hacein-Bey-Abina, Marina Cavazzana-Calvo, Alain Fischer

Blood (2012) 120 (9): 1957–1959.

https://doi.org/10.1182/blood-2012-04-426833

Published: 2012

Journal Articles

Major histocompatibility complex class II expression deficiency caused by a RFXANK founder mutation: a survey of 35 patients

Monia Ouederni, Quentin B. Vincent, Pierre Frange, Fabien Touzot, Sami Scerra, Mohamed Bejaoui, Aziz Bousfiha, Yves Levy, Barbara Lisowska-Grospierre, Danielle Canioni, Julie Bruneau, Marianne Debré, Stéphane Blanche, Laurent Abel, Jean-Laurent Casanova, Alain Fischer, Capucine Picard

Blood (2011) 118 (19): 5108–5118.

https://doi.org/10.1182/blood-2011-05-352716

Published: 2011

Includes: Supplemental data

Journal Articles

Clinical similarities and differences of patients with X-linked lymphoproliferative syndrome type 1 (XLP-1/SAP deficiency) versus type 2 (XLP-2/XIAP deficiency)

Blood (2011) 117 (5): 1522–1529.

https://doi.org/10.1182/blood-2010-07-298372

Published: 2011

Includes: Supplemental data

NARROW

Rac’n’rolling the immune system

Genotype, oxidase status, and preceding infection or autoinflammation do not affect allogeneic HCT outcomes for CGD

Inherited human Apollo deficiency causes severe bone marrow failure and developmental defects

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

Optimizing CD4+ T-Cells Transduction Protocol for Gene Therapy of HIV-1 Infected Patients

Reiterated Therapeutic Drug Monitoring (TDM) Dosing to Significantly Improve the Control of Exposure to IV Busulfan in Infants and Older Children Undergoing Hematopoietic Stem-Cell Transplantation (HSCT)

B Cell Reconstitution after Gene Therapy in Patients with Wiskott Aldrich Syndrome and Comparison with Mismatched Allogeneic Hematopoietic Stem Cell Transplantation

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

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

Faster T-cell development following gene therapy compared with haploidentical HSCT in the treatment of SCID-X1

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)

Circulating Endothelial Cells As a Reliable Marker Of Endothelial Damage In Children Undergoing Hematopoietic Stem Cell Transplantation

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

Anti CMV and/or Anti Adenovirus IFN-g-Positive CD4+ CD8+ T Lymphocytes for Treatment of Viral Infections After Allogeneic HSC Transplantation: First Results

Massive expansion of maternal T cells in response to EBV infection in a patient with SCID-Xl

Major histocompatibility complex class II expression deficiency caused by a RFXANK founder mutation: a survey of 35 patients

Clinical similarities and differences of patients with X-linked lymphoproliferative syndrome type 1 (XLP-1/SAP deficiency) versus type 2 (XLP-2/XIAP deficiency)

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American Society of Hematology

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American Society of Hematology

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