A novel gene therapy promoted transfusion independence in more than 90 percent of adult and pediatric patients with transfusion-dependent beta-thalassemia, according to a recent clinical trial published in The New England Journal of Medicine. The therapy represents a potentially curative treatment option for patients who must otherwise rely on life-long red blood cell transfusions. This approach provides a potential cure for patients with transfusion dependent beta-thalassemia without the need for an allogeneic donor, and these extremely encouraging results will give hope to patients otherwise not eligible for cure of their disease." Jennifer Schneiderman, MD, '06 MS, associate professor of Pediatrics in the Division of Hematology, Oncology and Stem Cell Transplantation and co-author of the study Alexis Thompson, MD, MPH, former section chief of Hematology in the Department of Pediatrics and associate director for Equity and Minority Health at the Robert H.
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