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Dr. Breda is developing safe, potentially curative genetic therapies for sickle cell disease and b-thalassemia.
Dr. Breda is the scientific co-director for the Comprehensive Center for the Cure of Sickle Cell Disease (SCD) and other RED Cell Disorders (CuRED) at CHOP. This program was created to provide a multidisciplinary care team and offer concrete curative therapies for patients affected by SCD and b-thalassemia (BT), two of the most frequent red cell disorders, also known as hemoglobinopathies. Dr. Breda has been developing safe genetic therapies for the cure of these conditions. The gene therapy approach represents a potential definitive cure for the patients, as it directly targets the underlining cause of the diseases. Dr. Breda engineered several lentiviral vectors, complex vehicles she engineered to carry and permanently transfer β-like globin genes in hosts cells. She established a platform to test how different red cells from patients, characterized by different mutations, respond to the various therapeutic vectors. She showed how patients' cells treated can recover hemoglobin production, in both SCD and BT. Within CuRED, Dr. Breda has been using animal models of BT and SCD and xenograft models, to demonstrate efficacy and safety of the genetic intervention. This preclinical evidence will be utilized to file an IND application for a clinical trial for hemoglobinopathies at CHOP.