Gudmundsdottir, Bjorg (NHLBI)
Tumburu, Laxminath (NHLBI)
The technology addresses treatment options for diseases such as sickle-cell and thalassemia. Traditionally, such beta-globinopathies 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. NIH inventors have identified a protein called Rio-Kinase 3 (RIOK3), that inhibits the production of fetal hemoglobin. Their work shows that inhibiting RIOK3 increases the production of fetal hemoglobin. Thus, RIOK3 is a promising novel therapeutic target to increase fetal hemoglobin expression.
- Designing lentiviral vectors to genetically target RIOK3
- Gene editing using endonucleases such as CRISPR/Cas9
- Developing orally administered RIOK3 specific kinase inhibitor drugs
- A novel and cost-effective treatment strategy in beta-globinopathies