Editing the core region in HPFH deletions alters fetal and adult globin expression for treatment of β-hemoglobinopathies
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Reactivation of fetal hemoglobin (HbF) is a commonly adapted strategy to ameliorate β-hemoglobinopathies. However, the continued production of defective adult hemoglobin (HbA) limits HbF tetramer production affecting the therapeutic benefits. Here, we evaluat... ...