University of North Carolina at Chapel Hill researchers have made a discovery that may have implications for the treatment of liver-based genetic defects such as hemophilia A and B in humans.
Mouse embryonic stem cells treated in culture with a growth factor and then injected into the liver reverse a form of hemophilia in mice analogous to hemophilia B in humans, the new study shows. A report of the study appears in the journal Proceedings of the National Academy of Sciences today (Feb. 15).
The genetically altered mice lack the clotting substance factor IX, which in humans results in the hereditary bleeding disorder known as hemophilia B. This disease, much less common than hemophilia A, affects roughly one of every 35,000 people, primarily males. Although embryonic stem, or ES, cells can differentiate into most cell types in the body, numerous problems have arisen in translating their potential into therapeutic strategies, the UNC School of Medicine study authors reported.
Leslie H. Lang | EurekAlert!
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