Development of inhibitors of RIOK3 for sickle cell anemia

National Center For Advancing Translational Sciences

Investigators

Abstract

Traditionally, beta-globinopathies such as sickle-cell anemia are treated through bone marrow transplantation. However, this method is limited due to high treatment costs and finding a matched-donor. This relies on increasing fetal hemoglobin to potentially cure the disease. The Tisdale Laboratory identified a protein called Rio-Kinase 3 (RIOK3) that inhibits the production of fetal hemoglobin. Inhibition of RIOK3 increases the production of fetal hemoglobin, suggesting that RIOK3 may be a promising novel therapeutic target to increase fetal hemoglobin expression. This project aims to use biophysical assays to identify a molecule capable of binding to RIOK3 to modulate its activity.