Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia
Of 35 patients with transfusion-dependent β-thalassemia treated with gene-edited autologous hematopoietic stem and progenitor cells, 32 had hemoglobin levels maintained without red-cell transfusions for at least 12 consecutive months.
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| Veröffentlicht in: | The New England journal of medicine 2024-05, Vol.390 (18), p.1663-1676 |
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| container_title | The New England journal of medicine |
| container_volume | 390 |
| creator | Locatelli, Franco Lang, Peter Wall, Donna Meisel, Roland Corbacioglu, Selim Li, Amanda M. de la Fuente, Josu Shah, Ami J. Carpenter, Ben Kwiatkowski, Janet L. Mapara, Markus Liem, Robert I. Cappellini, Maria Domenica Algeri, Mattia Kattamis, Antonis Sheth, Sujit Grupp, Stephan Handgretinger, Rupert Kohli, Puja Shi, Daoyuan Ross, Leorah Bobruff, Yael Simard, Christopher Zhang, Lanju Morrow, Phuong Khanh Hobbs, William E. Frangoul, Haydar |
| description | Of 35 patients with transfusion-dependent β-thalassemia treated with gene-edited autologous hematopoietic stem and progenitor cells, 32 had hemoglobin levels maintained without red-cell transfusions for at least 12 consecutive months. |
| doi_str_mv | 10.1056/NEJMoa2309673 |
| format | Article |
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| subjects | Adolescent Adolescent Medicine Adult Antigens, CD34 Autografts beta-Thalassemia - genetics beta-Thalassemia - therapy Blood diseases Blood Transfusion Bone marrow Busulfan Busulfan - therapeutic use CD34 antigen Cell therapy Child Childhood Diseases Collaboration CRISPR CRISPR-Cas Systems Europe Female Fetal Hemoglobin - biosynthesis Fetal Hemoglobin - genetics Fetuses Gene Editing Gene Editing - methods Genetics Genetics General Genome editing Genotypes Hematology Hematopoietic Stem Cell Transplantation - methods Hematopoietic Stem Cells Hemoglobin Hemopoiesis Humans Hypotheses Hypothesis testing Iron Leukocytes (neutrophilic) Male Mortality mRNA Myeloablative Agonists - therapeutic use Neutrophils North America Oncology Oncology General Pediatrics Pediatrics General Pharmaceuticals Progenitor cells Repressor Proteins - genetics Sickle cell disease Statistical analysis Stem Cells Teenagers Thalassemia Transplantation Conditioning Transplantation, Autologous Young Adult |
| title | Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia |
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