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Journal Articles

Ex Vivo Generation of Human T-Cell Progenitors from Cord Blood CD34+ Cells Can Accelerate Immune Reconstitution after Umbilical Cord Blood Transplantation in Adult Patient with Hematologic Malignancies

Blood (2024) 144 (Supplement 1): 7230.

https://doi.org/10.1182/blood-2024-210090

Published: 2024

Journal Articles

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

Raynier Devillier, Anne Huynh, Patrice Chevallier, Simona Piemontese, Regis Peffault de Latour, Patrizia Chiusolo, Aurelie Bauquet, Rahim Fandi, Sebastien Oster, Laura Simons, Isabelle Andre, Marina Cavazzana, Fabio Ciceri

Blood (2024) 144 (Supplement 1): 3457.

https://doi.org/10.1182/blood-2024-203203

Published: 2024

Journal Articles

Gene Therapy for Artemis-SCID and Artemis-Leaky-SCID Patients: Preliminary Results of the French Artegene Phase I/II Clinical Trial

Blood (2024) 144 (Supplement 1): 2201.

https://doi.org/10.1182/blood-2024-208350

Published: 2024

Journal Articles

Betibeglogene Autotemcel (beti-cel) Gene Addition Therapy Results in Durable Hemoglobin a Production with up to 10 Years of Follow-up with Transfusion-Dependent β-Thalassemia

Blood (2024) 144 (Supplement 1): 2194.

https://doi.org/10.1182/blood-2024-200152

Published: 2024

Journal Articles

Durable Clinical Benefits with Exagamglogene Autotemcel for Severe Sickle Cell Disease

Blood (2024) 144 (Supplement 1): 4954.

https://doi.org/10.1182/blood-2024-204001

Published: 2024

Journal Articles

Exagamglogene Autotemcel for Severe Sickle Cell Disease

Blood (2023) 142 (Supplement 1): 1052.

https://doi.org/10.1182/blood-2023-190139

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

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

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

Cryopreservation Impacts Allogenic Stem Cell Engraftment: Relation between Graft Quality Control and Post-Transplant Clinical Outcome

Clotilde Aussel, Jeromine Beaufort, Elisa Magrin, Olivia Leblanc, Laure Joseph, Francois Lefrere, Clemence Loiseau, Martin Castelle, Benjamin Fournier, Morseny Gueye, Despina Moshous, Alessandra Magnani, Marina Cavazzana, Felipe Suarez, Benedicte Neven, Ambroise Marçais, Jean-Sebastien Diana

Blood (2023) 142 (Supplement 1): 6949.

https://doi.org/10.1182/blood-2023-187478

Published: 2023

Journal Articles

Improvement in Iron Burden in Patients with Transfusion-Dependent β-Thalassemia (TDT) Treated with Betibeglogene Autotemcel (Beti-cel) Gene Therapy: Up to 9 Years of Follow-up

Blood (2023) 142 (Supplement 1): 2480.

https://doi.org/10.1182/blood-2023-173869

Published: 2023

Journal Articles

Sustained Efficacy, Safety, and Improved Quality of Life in Adult and Pediatric Patients with Transfusion-Dependent β-Thalassemia up to 9 Years Post Treatment with Betibeglogene Autotemcel (Beti-cel)

Blood (2023) 142 (Supplement 1): 1102.

https://doi.org/10.1182/blood-2023-174068

Published: 2023

Journal Articles

Adenine base editor–mediated correction of the common and severe IVS1-110 (G>A) β-thalassemia mutation

Giulia Hardouin, Panagiotis Antoniou, Pierre Martinucci, Tristan Felix, Sandra Manceau, Laure Joseph, Cécile Masson, Samantha Scaramuzza, Giuliana Ferrari, Marina Cavazzana, Annarita Miccio

Blood (2023) 141 (10): 1169–1179.

https://doi.org/10.1182/blood.2022016629

Published: 2023

Includes: Supplemental data

Journal Articles

Two Is Better Than One: Fetal Hemoglobin Reactivation and Alpha-Globin Downregulation to Correct the β-Hemoglobinopathy Phenotype through Base Editing

Panagiotis Antoniou, Mathieu von Joest, Letizia Fontana, Giulia Hardouin, Pierre Martinucci, Marina Cavazzana, Annarita Miccio

Blood (2022) 140 (Supplement 1): 7788–7789.

https://doi.org/10.1182/blood-2022-166330

Published: 2022

Journal Articles

Long Term Outcomes of 63 Patients with Transfusion-Dependent β-Thalassemia (TDT) Followed up to 7 Years Post-Treatment with betibeglogene autotemcel (beti-cel) Gene Therapy and Exploratory Analysis of Predictors of Successful Treatment Outcomes in Phase 3 Trials

Blood (2022) 140 (Supplement 1): 5355–5357.

https://doi.org/10.1182/blood-2022-162400

Published: 2022

Journal Articles

Long-Term Patient-Reported Outcomes Following Treatment with betibeglogene autotemcel in Patients with Transfusion-Dependent β-Thalassemia

Franco Locatelli, Mark C. Walters, Janet L. Kwiatkowski, Marina Cavazzana, Suradej Hongeng, John B. Porter, Adrian J. Thrasher, Andreas E. Kulozik, Isabelle Thuret, John E.J. Rasko, Evangelia Yannaki, Martin G. Sauer, Shamshad Ali, Himal Thakar, Katiana Gruppioni, Alexis A. Thompson

Blood (2022) 140 (Supplement 1): 8200–8202.

https://doi.org/10.1182/blood-2022-163178

Published: 2022

Journal Articles

Targeted Base Editing Strategies for Beta-Hemoglobinopathies

Giulia Hardouin, Panagiotis Antoniou, Pierre Martinucci, Tristan Felix, Marta Zoccheddu, Anne Chalumeau, Giacomo Frati, Sandra Manceau, Laure Joseph, Oriana Romano, Marina Cavazzana, Annarita Miccio

Blood (2022) 140 (Supplement 1): 7778–7779.

https://doi.org/10.1182/blood-2022-162362

Published: 2022

Journal Articles

Four decades of progress

Marina Cavazzana

Blood (2022) 140 (7): 665–666.

https://doi.org/10.1182/blood.2022017211

Published: 2022

Journal Articles

Hematopoietic stem cell transplantation for Wiskott-Aldrich syndrome: an EBMT Inborn Errors Working Party analysis

Blood (2022) 139 (13): 2066–2079.

https://doi.org/10.1182/blood.2021014687

Published: 2022

Includes: Supplemental data

Journal Articles

Novel Lentiviral Vectors for Gene Therapy of Sickle Cell Disease Combining Gene Addition and Gene Silencing Strategies

Megane Brusson, Anne Chalumeau, Pierre Martinucci, Valentina Poletti, Fulvio Mavilio, Marina Cavazzana, Mario Amendola, Annarita Miccio

Blood (2021) 138 (Supplement 1): 3973.

https://doi.org/10.1182/blood-2021-151076

Published: 2021

NARROW

Ex Vivo Generation of Human T-Cell Progenitors from Cord Blood CD34+ Cells Can Accelerate Immune Reconstitution after Umbilical Cord Blood Transplantation in Adult Patient with Hematologic Malignancies

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

Gene Therapy for Artemis-SCID and Artemis-Leaky-SCID Patients: Preliminary Results of the French Artegene Phase I/II Clinical Trial

Betibeglogene Autotemcel (beti-cel) Gene Addition Therapy Results in Durable Hemoglobin a Production with up to 10 Years of Follow-up with Transfusion-Dependent β-Thalassemia

Durable Clinical Benefits with Exagamglogene Autotemcel for Severe Sickle Cell Disease

Exagamglogene Autotemcel for Severe Sickle Cell Disease

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

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

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

Cryopreservation Impacts Allogenic Stem Cell Engraftment: Relation between Graft Quality Control and Post-Transplant Clinical Outcome

Improvement in Iron Burden in Patients with Transfusion-Dependent β-Thalassemia (TDT) Treated with Betibeglogene Autotemcel (Beti-cel) Gene Therapy: Up to 9 Years of Follow-up

Sustained Efficacy, Safety, and Improved Quality of Life in Adult and Pediatric Patients with Transfusion-Dependent β-Thalassemia up to 9 Years Post Treatment with Betibeglogene Autotemcel (Beti-cel)

Adenine base editor–mediated correction of the common and severe IVS1-110 (G>A) β-thalassemia mutation

Two Is Better Than One: Fetal Hemoglobin Reactivation and Alpha-Globin Downregulation to Correct the β-Hemoglobinopathy Phenotype through Base Editing

Long Term Outcomes of 63 Patients with Transfusion-Dependent β-Thalassemia (TDT) Followed up to 7 Years Post-Treatment with betibeglogene autotemcel (beti-cel) Gene Therapy and Exploratory Analysis of Predictors of Successful Treatment Outcomes in Phase 3 Trials

Long-Term Patient-Reported Outcomes Following Treatment with betibeglogene autotemcel in Patients with Transfusion-Dependent β-Thalassemia

Targeted Base Editing Strategies for Beta-Hemoglobinopathies

Four decades of progress

Hematopoietic stem cell transplantation for Wiskott-Aldrich syndrome: an EBMT Inborn Errors Working Party analysis

Novel Lentiviral Vectors for Gene Therapy of Sickle Cell Disease Combining Gene Addition and Gene Silencing Strategies

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American Society of Hematology

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ASH Publications

American Society of Hematology

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