Leaders in Research
Designing the next generation of curative therapies
CuRED is proud to be a part of the research that is leading to cures for sickle cell disease, beta thalassemia, and other red cell disorders. In the Rivella lab, our scientists are working to develop a gene therapy to cure sickle cell disease and other red cell disorders.
In patients with sickle cell disease and beta-thalassemia, hemoglobin, or the molecule that moves oxygen through your body, is not produced at adequate levels. This is caused by a mutation in the gene that produces hemoglobin. Our researchers are building living drugs, known as gene therapies, that will deliver corrective versions of these genes to patient cells. The lab is testing these therapies in mice and expect to launch a clinical trial to bring this kind of research to patients at CHOP in the next several years. Learn more about the Rivella lab’s research on gene therapies for hemoglobinopathies here.
Our physician-scientists are also working to develop a better understanding of these diseases and potential other treatment options, including bone marrow transplants and fertility preservation methods.
Our areas of focus
Our labs are also investigating many methods to improve clinical outcomes for patients! Our research focuses on the following:
- Curative therapies for hemoglobinopathies and other red cell disorders
- Improving outcomes for patients following a hematopoietic stem cell transplant
- Fertility preservation options
- Therapies to increase red cell production and prevent iron overload in beta-thalassemia patients
The division’s location within a hospital environment provides valuable resources to conducting successful clinical research.
- The Children's Hospital of Philadelphia Research Institute is a facility specifically dedicated to the administration of all research conducted at CHOP.
- CHOP’s Institutional Review Board (IRB) ensures the protection of subjects in compliance with federal law.
- The clinical laboratory facilities at CHOP are state accredited by the College of American Pathologists and certified by the Department of Health and Human Services.
Clinical research finder tool
To learn more about the current and ongoing clinical trials at CHOP, please use our Clinical Research Finder tool. When accessing the tool, please use as many search terms as possible to learn about various studies.
If you are interested in learning about new opportunities to participate in research, you can also sign up for our research participant registry.