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

8 Sept 2020

Abstract excerpt

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 induction potential of erythroid-specific BCL11A enhancer-edited HSPCs in 4 nonhuman primates. We used a single guide...

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PMID: 32897878
DOI: 10.1172/jci140189

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