Research at The Childrens Hospital of Philadelphia may advance treatment of the blood disease thalassemia
Hematology researchers at The Children’s Hospital of Philadelphia have discovered a gene and its associated protein that may have major implications for red blood cell formation, specifically for hemoglobin, which carries oxygen in red blood cells. Understanding how this protein functions may eventually lead to novel treatments for the hemoglobin-related blood disease, thalassemia.
Thalassemia is a group of related inherited disorders that together are the most common single-gene disease known. The most severe form of the disease, beta thalassemia major, affects 300,000 patients worldwide.
John Ascenzi | EurekAlert
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