Sickle Cell Disease Program | Research & Innovation

In the setting of one of the world’s most extensive pediatric research hospitals, we apply a science-driven approach to sickle cell disease. Dana-Farber/Boston Children's was one of the original National Institutes of Health-funded comprehensive sickle cell centers in the United States, and a site for the Sickle Cell Disease Clinical Research Network. Our researchers conduct clinical and laboratory studies to increase and enhance knowledge of sickle cell disorders and treatment. Keeping abreast of current basic scientific research, its translation to clinical care, and advising families and referring physicians about available clinical trials are among our highest priorities.

Gene therapy boosts fetal hemoglobin

A pilot gene therapy treatment for sickle cell disease, restoring patients’ ability to make fetal hemoglobin, has produced good results in the first three patients.

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Doctor examining a patient who was treated using a gene therapy for sickle cell disease,
In the 1980s, our physicians were the first to recognize the potential of hydroxyurea to treat sickle cell disease and to use the medication in children. Today, our researchers continue to work on developing new methods of diagnosing and treating sickle cell disease, including stem cell researchstem cell transplantation, and gene therapy. Search for current sickle cell disease clinical trials.