Long-term efficacy and safety results of betibeglogene autotemcel gene therapy for transfusion-dependent β-thalassemia
J. Kwiatkowski, Alexis A. Thompson, Jennifer Schneiderman, Isabelle Thuret et autres
6 citationsBlood
Himal L. Thakar
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14Publications signalées
303Citations signalées
1Affiliations récentes
Institutions déclarées
Domaines associés
Hemoglobinopathies and Related DisordersAcute Lymphoblastic Leukemia researchPeroxisome Proliferator-Activated ReceptorsVirus-based gene therapy researchNeuroblastoma Research and Treatments
Publications récentes
Betibeglogene Autotemcel (Beti-cel) Gene Addition Therapy Results in Durable Hemoglobin A (HbA) Production with up to 10 Years of Follow-up in Participants with Transfusion-Dependent β-Thalassemia
Timothy S. Olson, Alexis A. Thompson, Janet L. Kwiatkowski, Jennifer Schneiderman et autres
2 citationsTransplantation and Cellular Therapy
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
Alexis A. Thompson, Janet L. Kwiatkowski, Jennifer Schneiderman, Isabelle Thuret et autres
3 citationsBlood
Betibeglogene autotemcel gene therapy in patients with transfusion-dependent, severe genotype β-thalassaemia (HGB-212): a non-randomised, multicentre, single-arm, open-label, single-dose, phase 3 trial
Janet L. Kwiatkowski, Mark C. Walters, Suradej Hongeng, Evangelia Yannaki et autres
43 citationsThe Lancet
Gene therapy in transfusion-dependent non-β0/β0 genotype β-thalassemia: first real-world experience of beti-cel
Adil Mirza, Mona-Lisa Ritsert, Gloria Tao, Himal L. Thakar et autres
12 citationsBlood Advances
Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy
Christine Duncan, Jacob R. Bledsoe, Bartosz Grzywacz, Amy Beckman et autres
139 citationsNew England Journal of Medicine
Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy
Florian Eichler, Christine Duncan, Patricia L. Musolino, Troy C. Lund et autres
81 citationsNew England Journal of Medicine
Secondary failure of lentiviral vector gene therapy in a cerebral adrenoleukodystrophy patient with an ABCD1 whole-gene deletion
Troy C. Lund, Paul J. Orchard, David Nascene, Carina King et autres
7 citationsMolecular Therapy
Evaluation of Mobilization, Apheresis, and Conditioning Regimen and Engraftment in Patients Receiving One-Time Gene Therapy with Elivaldogene Autotemcel (Eli-cel) for Cerebral Adrenoleukodystrophy (CALD)
Christine Duncan, Paul J. Orchard, Florian Eichler, Jörn‐Sven Kühl et autres
2 citationsTransplantation and Cellular Therapy
Sustained Efficacy and Safety in Adult and Pediatric Patients with Transfusion-Dependent β-Thalassemia up to 9 Years Post Treatment with Betibeglogene Autotemcel (Beti-cel)
Mark C. Walters, Alexis A. Thompson, Timothy S. Olson, John B. Porter et autres
1 citationTransplantation and Cellular Therapy
Mobilization and Apheresis Experience in Patients Receiving One-Time Gene Therapy with Beti-Cel for Patients with Transfusion-Dependent β-Thalassemia: A Pooled Analysis
Jennifer Schneiderman, David F. Friedman, Christina Anderson, Ge Tao et autres
0 citationsTransplantation and Cellular Therapy
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)
Alexis A. Thompson, Timothy S. Olson, Mark C. Walters, John B. Porter et autres
6 citationsBlood
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