Genome editing of HBG1 and HBG2 to induce fetal hemoglobin
Abstract
Key Points Cas9 editing of the γ-globin gene promoters in hematopoietic stem cells (HSCs) increases red cell HbF by ≤40%. No deleterious effects on hematopoiesis or off-target mutations were detected 16 weeks after xenotransplantation of edited...
Paper Details
Title
Genome editing of HBG1 and HBG2 to induce fetal hemoglobin
Published Date
Nov 6, 2019
Journal
Volume
3
Issue
21
Pages
3379 - 3392
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Notes
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