Kwame Ohene-Frempong Chair on Sickle Cell Anemia
Children's hospital of Philadelphia
Philadelphia, Pennsylvania
Stefano Rivella, PhD, is a research faculty member in the Division of Hematology at Children's Hospital of Philadelphia. He holds the Kwame Ohene-Frempong Endowed Chair in Pediatric Hematology.
Dr. Rivella is an expert in the pathophysiology of erythroid and iron disorders and in the generation of lentiviral vectors for the cure of hemoglobinopathies. He also investigates additional disorders such as anemia of inflammation and hemochromatosis. Specifically, he generated the first adult mouse model of ß-thalassemia major and contributed to the creation of the first lentiviral vector that corrected the hemoglobin synthesis in mice affected by ß-thalassemia. This led the way to gene therapy clinical trials. He is also generating new lentiviral vectors for the treatment of ß-thalassemia and sickle cell anemia. He showed correction of the hemoglobin synthesis defect in mice as well as in patient cells in vitro, as a preclinical test for potential gene therapy trials. In addition, Dr. Rivella investigates disorders like anemia of inflammation, which is a form of anemia associated with many inflammatory disorders, and hemochromatosis, which is associated with abnormal iron absorption from the diet. He also studies polycythemia vera, which is associated with an excessive production of red cells. Dr. Rivella has been providing expertise on mouse models important for red blood cell and macrophage biology as well as inflammation and iron metabolism to many U.S. laboratories and pharmaceutical companies interested in learning these subjects or testing new drugs. His team is leaders in the discovery of erythroid and iron disorders including beta-thalassemia. The team has made significant contributions to the scientific body of literature regarding red blood cells, and how manipulating the production of red cells can lead to the development of new disease-modifying therapies. The Rivella Lab works in tandem with the Comprehensive Center for the Cure of Sickle Cell Disease and Other Red Blood Cell Disorders (CuRED), a CHOP-designated Frontier Program that aims to develop and deliver novel gene therapies and stem cell transplants to patients in a family-centered clinic.
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Saturday, October 25, 2025
1:45 PM - 2:45 PM PT