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.