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Transplanted Liver Cells Correct Hemophilia A in Mice

On February 14, 2008, an international team of researchers published the results of studies demonstrating the successful restoration of factor VIII (FVIII) production, the missing clotting protein, in mice with hemophilia A. The principal investigator of the study was Sanjeev Gupta, MD, MB, BS, Professor of Hepatology at Albert Einstein College of Medicine in New York.
By transplanting functioning endothelial cells in the livers of healthy mice to the livers of mice with hemophilia, the team was able to stimulate FVIII production to levels that corrected previously uncontrolled bleeding. Endothelial cells line blood vessels and organs in the circulatory system. They provide an anticoagulant barrier between the vessel wall and blood. Gupta reported that three months following transplantation, the number of healthy endothelial cells in the mice had increased enough to trigger the production of FVIII in amounts that corrected their hemophilia. 

Past research has shown that endothelial cells, once thought to be limited in function and adaptability, are actually capable of performing a number of important roles. The findings made by Gupta and colleagues appear to further validate this conclusion. Although it is known that FVIII is manufactured in the liver, the particular cells responsible for its production were still to be determined. The targeted use of the liver’s endothelial cells in this research, therefore, represents a research breakthrough.

The next step, Gupta explained, is to study how this therapeutic approach could be developed for humans. “From the treatment point of view, we can now begin to direct our attention to these particular cells and find the most effective way to cure this disorder in people,” he said. Gupta also suggested that the same protocol could potentially be used in hemophilia B, or factor IX deficiency.
The study, “Transplanted Endothelial Cells Repopulate the Liver Endothelium and Correct the Phenotype of Hemophilia A Mice,” was published in the February 14, 2008, online issue of The Journal of Clinical Investigation.

Source: The Washington Post, February 14, 2008


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