Results for Hemoglobinopathies

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.

Dr. Freedman has a particular interest in epidemiologic and translational research within pediatric supportive oncology. He aims to advance the understanding and treatment of symptoms, infections, and organ toxicities in pediatric cancer and hematopoietic stem cell transplant patients.

Dr. Kwiatkowski is director of the Thalassemia Center at CHOP. Her research focuses on gene therapy for hemoglobinopathies, monitoring and treatment of complications of thalassemia, sickle cell disease, pyruvate kinase deficiency, and monitoring and treatment of transfusional iron overload.

Dr. Flake is a general pediatric surgeon with a clinical and research focus on prenatal treatment ranging from the fetal surgical repair of anatomic anomalies to prenatal stem cell and gene therapy. He has extensive experience in developing rodent, canine, and sheep models for in utero transplantation and for investigating fetal surgery for anatomic malformations.

Dr. Abdulmalik’s research interests involve hemoglobinopathies and red blood cell disorders, with a focus on sickle cell disease and beta-thalassemia.

Dr. Breda is developing safe, potentially curative genetic therapies for sickle cell disease and b-thalassemia.

Dr. Guerra is researching innovating therapies to treat β-thalassemia and sickle cell anemia and use lentiviruses as a tool. Her work aims to discover new biological targets that can be used to treat β-thalassemia and other forms of anemia.