Editing the core region in HPFH deletions alters fetal and adult globin expression for treatment of β-hemoglobinopathies
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 evaluated deletional hereditary persistence of f...
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Elsevier,
2023-06-01T00:00:00Z.
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