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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Bibliographische Detailangaben
Veröffentlicht in:	The New England journal of medicine 2024-05, Vol.390 (18), p.1663-1676
Hauptverfasser:	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
Format:	Artikel
Sprache:	eng
Schlagworte:	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
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Beschreibung
Zusammenfassung:	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.
ISSN:	0028-4793 1533-4406 1533-4406
DOI:	10.1056/NEJMoa2309673