Preliminary Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta Thalassemia
Smith A, Schiller G, Vercellotti G, Kwiatkowski J, Krishnamurti L, Esrick E, Williams D, Miller W, Woolfson A, Walters M. Preliminary Results of a Phase 1/2 Clinical Study of Zinc Finger Nuclease-Mediated Editing of BCL11A in Autologous Hematopoietic Stem Cells for Transfusion-Dependent Beta Thalassemia. Blood 2019, 134: 3544. DOI: 10.1182/blood-2019-125743.Peer-Reviewed Original ResearchTransfusion-dependent beta thalassemiaPeripheral blood mononuclear cellsSerious adverse eventsHematopoietic stem cell transplantationHuman hematopoietic stem cellsHematopoietic reconstitutionBluebird BioPRBC transfusionPatient 1HbF levelsG-CSFJazz PharmaceuticalsPacked red blood cell transfusionPhase I/II studyAllogeneic hematopoietic stem cell transplantationRed blood cell transfusionHematopoietic stem cellsPhase 1/2 clinical studyBeta thalassemiaInvestigational cell therapySpeakers bureauBlood cell transfusionStem cell transplantationBlood mononuclear cellsCells/