Integrative microRNA and gene expression analysis identifies new drug repurposing candidates for fetal hemoglobin induction in β-hemoglobinopathies

Abstract Therapeutic inductionof fetal hemoglobin(HbF) is one of the most promising approaches to ameliorate the severity of hemoglobinopathieslike β-thalassemia and sickle cell anemia. Although several pharmacological agents have been investigated for HbF inductionin adults, the majority of these are associated with significant side-effects. While drug repurposingis known to open new doors for the use of approved drugsin unexplored clinical conditions, the primary challenge lies in identifying such candidates. In this study, we aimed to identify repurposingcandidates for HbF inductionusing a novel in silico approach utilizing microRNA-pathway-drug relationships. A computational drug repurposingstrategy identified several unique candidates for HbF induction; among which Curcumin, Ginsenoside, Valproate, and Vorinostatwere found to be most suitable for future trials. This study identified new drug repurposingcandidates for HbF inductionand demonstrates an easily adaptable methodology that can be used for other pathophysiological conditions.