Fetal hemoglobin in β hemoglobinopathies: Is enough too much?

Abstract excerpt

Cell-based therapies inducing about 40% fetal hemoglobin (HbF), or a HbF-like hemoglobin in most erythrocytes, can—at least in the short-term—effect a cure or near-cure of β hemoglobinopathies, which are humankind's most common Mendelian diseases.1-3 In sickle cell disease, a point mutation in the normal β-globin gene (HBB) directs the synthesis of sickle hemoglobin (HbS), which polymerizes on deoxygenation,...

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