Platform for the prediction of HbF inducers

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Description

Pharmacological reactivation of the γ-globin gene for the production of fetal hemoglobin (HbF) is a very promising therapeutic avenue for β-thalassemia. Increased production of γ-globin can ameliorate the symptoms of the disease by partly substituting the non-functional β-globin gene and restoring the balance between α and non-α globin chains. Drugs currently available for this purpose have limited application due to moderate therapeutic properties, variable patient response and potential cytotoxic effects. Although research into finding new HbF-inducing agents is imperative, the absence of a known biological target hampers drug discovery.

This work was supported by funding under the Seven Research Framework Programme of the European Union. Project THALAMOSS (HEALTH.2012.1.2-1 Grant agreement no: 306201).