Enzyme Identified as Possible Novel Drug Target for Sickle Cell Disease, Thalassemia
Children's Hospital of PhiladelphiaMedical researchers have identified a key signaling protein that regulates hemoglobin production in red blood cells, offering a possible target for a future innovative drug to treat sickle cell disease. Tests in human cells reveal that blocking the protein reduces the characteristic sickling that distorts the shape of red blood cells and gives the disease its name.