Disorders of adult beta-globin synthesis, which include sickle cell disease (SCD) and beta-thalassemia, are the most common monogenic disorders in the world. While the curative potential of bone marrow transplantation has been demonstrated, this approach is limited to a small fraction of affected patients due to the requirement for an HLA-matched donor, the highly specialized approach that requires critical infrastructure, and the high cost.
Scientists at the National Heart, Lung, and Blood Institute (NHLBI), have developed lentiviral vectors encoding shRNA to Rio-Kinase 3 (RIOK3) that decrease expression of RIOK3, leading to more robust fetal hemoglobin expression.