Biologic and Clinical Efficacy of LentiGlobin for Sickle Cell Disease

An unprespecified interim analysis of results from a phase 1–2 study of gene therapy for sickle cell disease shows resolution of severe vaso-occlusive events in 25 patients who could be evaluated. In the 24 months before enrollment, these patients had a median of 3.5 severe vaso-occlusive events per...

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Veröffentlicht in:	The New England journal of medicine 2022-02, Vol.386 (7), p.617-628
Hauptverfasser:	Kanter, Julie, Walters, Mark C, Krishnamurti, Lakshmanan, Mapara, Markus Y, Kwiatkowski, Janet L, Rifkin-Zenenberg, Stacey, Aygun, Banu, Kasow, Kimberly A, Pierciey, Francis J, Bonner, Melissa, Miller, Alex, Zhang, Xinyan, Lynch, Jessie, Kim, Dennis, Ribeil, Jean-Antoine, Asmal, Mohammed, Goyal, Sunita, Thompson, Alexis A, Tisdale, John F
Format:	Artikel
Sprache:	eng
Schlagworte:	Adolescent Adult Anemia Anemia, Sickle Cell - blood Anemia, Sickle Cell - complications Anemia, Sickle Cell - therapy Apheresis Autografts beta-Globins - genetics Bone marrow Child Enrollments Female Fetal Hemoglobin Gene therapy Genetic Therapy Genetic Vectors Genetics Genetics General Graft versus host disease Hematology Hematopoietic Stem Cell Transplantation Hematopoietic stem cells Hemoglobin Hemoglobins - analysis Hemoglobins - genetics Hemoglobins - metabolism Hemolysis Humans Lentivirus Male Middle Aged Oncology Oncology General Patients Progenitor cells Sickle cell disease Stem Cell Transplantation Transplantation Transplants & implants Vascular Patency Young Adult
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Beschreibung
Zusammenfassung:	An unprespecified interim analysis of results from a phase 1–2 study of gene therapy for sickle cell disease shows resolution of severe vaso-occlusive events in 25 patients who could be evaluated. In the 24 months before enrollment, these patients had a median of 3.5 severe vaso-occlusive events per year.
ISSN:	0028-4793 1533-4406
DOI:	10.1056/NEJMoa2117175