Medicinal Chemistry of Fetal Hemoglobin Inducers for Treatment of β-Thalassemia

Abstract excerpt

In this review we summarize the achievements of medicinal chemistry in the field of pharmacological approaches to the therapy of beta-thalassemia using molecules able to stimulate the production of fetal hemoglobin (HbF). We first describe the molecular basis of the pathology and the biochemical rational of using HbF inducers for therapy; we then outlined the in vitro and in vivo experimental systems suitable for...

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