Dr. Laura Breda
Dr. Breda’s work is focused on developing genetic therapies for the cure of conditions such as Sickle Cell Disease (SCD) and β-Thalassemia, also known as hemoglobinopathies. At the moment, patients with SCD and β-Thalassemia undergo palliative care to overcome anemia and the multiple comorbidities acquired over time. The gene therapy approach, instead, represents a potential definitive cure for the patients, as it targets directly the underlining cause of the diseases. Dr. Breda developed several gene therapy tools to restore the production of hemoglobin, the oxygen carrier protein that is insufficient or dysfunctional in the red cells of patients with hemoglobinopathies. By using lentiviral vectors, complex vehicles she engineered to carry and permanently transfer β-like globin genes in hosts cells, she showed how patients’ cells treated can recover hemoglobin production, in both SCD and β-Thalassemia. She developed a platform to test in the laboratory how different red cells from patients, characterized by different mutations, can have different responses to the various constructs developed. This laboratory step is pivotal for developing safe and effective therapies for future clinical trials.