Scientists at the Albert Einstein College of Medicine of Yeshiva University have shown for the first time that transplanted cells can cure hemophilia A (the most common form of the disease) in an animal model. Their study appears online February 14 in the Journal of Clinical Investigation.
Hemophilia A affects one in 10,000 males and is characterized by recurrent, spontaneous bleeding that can cause disability or death. It has, most famously, affected British and Russian nobility, along with countless others. The Einstein team, led by Dr. Sanjeev Gupta, cured hemophilia A by transplanting healthy liver endothelial cells from donor mice into a mouse model of the disease. In curing these mice, the Einstein researchers also overturned conventional wisdom regarding which cells produce factor VIII, the crucial clotting protein that is lacking in people with type A hemophilia.
Weve known for three decades that factor VIII is produced in the liver, but precisely where has been controversial, says Dr. Gupta, a professor in the departments of medicine and pathology at Einstein. It was assumed that factor VIII was made by the hepatocytescells that perform many of the livers functions and comprise most of its bulk. But our research had suggested that the primary sources were special endothelial cells that line the sinusoids, the livers blood-filled spaces. We did this study to confirm the role of liver endothelial cells in producing factor VIII and to see if transplanting them from a healthy donor liver could correct hemophilia A in an animal model.
The donor mice used by Dr. Guptas team had been genetically engineered so that their endothelial cells expressed the gene for green fluorescent protein. This allowed the researchers to see whether the liver endothelial cellsisolated from donor livers and then injected into a vein leading into the liver of recipient hemophilia A micecould successfully engraft within the recipients livers. Before tran
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Albert Einstein College of Medicine