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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

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

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

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

Journal Articles

Clinical Results of the Drepaglobe Trial for Sickle Cell Disease Patients

Blood (2021) 138 (Supplement 1): 1854.

https://doi.org/10.1182/blood-2021-152331

Published: 2021

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

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

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

Restoring Iron Homeostasis in Pts Who Achieved Transfusion Independence after Treatment with Betibeglogene Autotemcel Gene Therapy: Results from up to 7 Years of Follow-up

Alexis A. Thompson, Franco Locatelli, Evangelia Yannaki, Mark C. Walters, John B. Porter, Suradej Hongeng, Andreas E. Kulozik, Martin G. Sauer, Adrian J. Thrasher, Isabelle Thuret, Ashutosh Lal, Marina Cavazzana, John E.J. Rasko, Lin Du, Richard A. Colvin, Janet L. Kwiatkowski

Blood (2021) 138 (Supplement 1): 573.

https://doi.org/10.1182/blood-2021-148177

Published: 2021

NARROW

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)

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

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

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

Clinical Results of the Drepaglobe Trial for Sickle Cell Disease Patients

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

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

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

Restoring Iron Homeostasis in Pts Who Achieved Transfusion Independence after Treatment with Betibeglogene Autotemcel Gene Therapy: Results from up to 7 Years of Follow-up

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American Society of Hematology

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ASH Publications

American Society of Hematology

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