BCL11A enhancer-edited hematopoietic stem cells persist in rhesus monkeys without toxicity

Gene editing of the erythroid-specific BCL11A enhancer in hematopoietic stem and progenitor cells (HSPCs) from patients with sickle cell disease (SCD) induces fetal hemoglobin (HbF) without detectable toxicity, as assessed by mouse xenotransplant. Here, we evaluated autologous engraftment and HbF in...

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Veröffentlicht in:	The Journal of clinical investigation 2020-12, Vol.130 (12), p.6677-6687
Hauptverfasser:	Demirci, Selami, Zeng, Jing, Wu, Yuxuan, Uchida, Naoya, Shen, Anne H, Pellin, Danilo, Gamer, Jackson, Yapundich, Morgan, Drysdale, Claire, Bonanno, Jasmine, Bonifacino, Aylin C, Krouse, Allen E, Linde, Nathaniel S, Engels, Theresa, Donahue, Robert E, Haro-Mora, Juan J, Leonard, Alexis, Nassehi, Tina, Luk, Kevin, Porter, Shaina N, Lazzarotto, Cicera R, Tsai, Shengdar Q, Weiss, Mitchell J, Pruett-Miller, Shondra M, Wolfe, Scot A, Bauer, Daniel E, Tisdale, John F
Format:	Artikel
Sprache:	eng
Schlagworte:	Autografts Binding sites Biomedical research Blood Bone marrow Erythrocytes Erythropoiesis Fetuses GATA-1 protein Genome editing Genomes Hematopoietic stem cells Hemoglobin Peripheral blood Progenitor cells Proteins Ribonucleic acid RNA Sickle cell disease Stem cell transplantation Stem cells Toxicity Transplants & implants Xenografts
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