Discovery of Novel Fetal Hemoglobin Inducers through Small Chemical Library Screening.

摘要: The screening of chemical libraries based on cellular biosensors is a useful approach to identify new hits for novel therapeutic targets involved in rare genetic pathologies, such as β-thalassemia and sickle cell disease. In particular, pharmacologically mediated stimulation human γ-globin gene expression, increase fetal hemoglobin (HbF) production, have been suggested potential strategies these hemoglobinopathies. this article, we screened small library, constituted 150 compounds, using the biosensor K562.GR, carrying enhanced green fluorescence protein (EGFP) red (RFP) genes under control β-globin promoters, respectively. Then identified compounds were analyzed HbF inducers primary cultures, obtained from patients, confirming their activity inducers, suggesting molecules lead further biological investigations.