Research

Sickle cell disease (SCD) is a complex genetic condition that affects millions of people worldwide, causing progressive organ damage, severe anemia, and shortened lifespan. Our researchers are investigating the cellular and molecular drivers of complications such as kidney injury, vascular dysfunction, and neurological impairment. By studying how hemolysis, inflammation, and vascular changes contribute to organ damage, investigators are identifying new biomarkers and therapeutic targets with the potential to improve outcomes. Their work spans from laboratory studies, such as evaluating HIV reservoirs and mitochondrial dysfunction, to clinical trials exploring innovative treatments and prevention strategies, with the goal of developing both novel therapies and improved approaches to risk assessment.

In addition to basic and translational research, the division is deeply engaged in global efforts to reduce the burden of SCD through international collaborations, particularly in sub-Saharan Africa where the disease is most common. Clinical trials in both Africa and the United States are exploring cost-effective treatments for anemia and other chronic complications, while new partnerships are helping to expand access to advanced diagnostic testing. Together, these initiatives reflect a comprehensive approach to SCD, linking laboratory discoveries with real-world interventions to improve care and quality of life for patients around the world.

Hemophilia and bleeding disorders research within the division is dedicated to advancing diagnosis, treatment, and quality of life for people with inherited bleeding conditions. Efforts focus on hemophilia and von Willebrand disease (VWD), with particular attention to women’s health concerns such as heavy menstrual bleeding, iron deficiency, and postpartum bleeding. Researchers also lead and participate in multi-center trials testing new and emerging therapies, including extended half-life factor products, gene therapy, siRNA, and monoclonal antibodies, while exploring inhibitor prevention, the impact of aging on bleeding disorders, and genotype-phenotype relationships in VWD. By developing integrated care models and addressing barriers to care, the program seeks to improve early recognition and individualized treatment.

Research on iron metabolism focuses on how cells regulate and use iron to meet their functional needs. Investigators are examining how mitochondrial health interacts with iron metabolism, how iron influences cellular development in tissues that require high levels of iron and heme (such as the liver and red blood cell–producing tissues), and how these processes adapt during pregnancy. This work aims to deepen understanding of iron’s role in health and disease, with the potential to guide new approaches to managing iron-related disorders.

Ongoing thrombosis and platelet disorder research focuses on the fundamental mechanisms that drive clot formation, platelet activation, and vascular injury, as well as strategies to improve diagnosis and treatment of related diseases. Using advanced imaging technologies, investigators are uncovering how platelets interact with blood vessels, immune cells, and clotting factors in both healthy and disease states, including sickle cell disease. Other studies focus on platelet mitochondria as biomarkers of human health and disease, offering new ways to assess bioenergetics and disease progression across diverse patient populations. Clinically, the program leads and participates in national trials targeting thrombotic microangiopathies such as thrombotic thrombocytopenic purpura (TTP), while also developing and testing novel therapies, including immunotherapies and monoclonal antibodies. Together, these efforts are advancing understanding of platelet biology and thrombosis to inform more effective, personalized approaches to patient care.