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Redesigned protein accelerates blood clotting
Date:12/22/2008

Researchers have made several, subtle changes in the structure of a key protein, dramatically increasing its ability to drive blood clotting, according to a study published in a December edition of the Journal of Thrombosis and Haemostasis. The findings have profound implications for the treatment of hemophilia, the inherited blood disorder that causes easy or excessive bleeding in 30,000 Americans.

In most cases, hemophilia is caused by a lack of a protein, factor VIII, one of several proteins in a chain reaction that enables blood to solidify, or clot, to plug wounds after injury. Current preventive treatment consists of genetically engineered factor VIII administered by injection, but one quarter of those born with no factor VIII suffer severe immune reactions that render the treatment inactive. In addition, current treatment costs as much as $200,000 per patient per year. Researchers at the University of Rochester Medical Center have been studying the structure and function of factor VIII for decades, and are now making subtle changes in the protein with the goal of offering more effective, less burdensome treatment.

In past research and in the current study, a team led by Philip Fay, Ph.D., professor in the Department of Biochemistry and Biophysics at the University of Rochester Medical Center, has identified key amino acids (out of the more than 2,300 building blocks making up factor VIII) with the potential, if replaced, to change the performance of entire protein. Fay, along with partner, Hironao Wakabayashi, M.D., research assistant professor, has filed patent applications for the factor VIII redesigns.

"Our goal is to improve upon nature by developing gain-of-function factor VIII proteins that are superior to the version of the protein found in healthy individuals," Fay said. "These more potent forms are not likely to occur naturally since they would theoretically result in excessive clotting, blocked arteries
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Contact: Greg Williams
Greg_Williams@urmc.rochester.edu
585-275-3676
University of Rochester Medical Center
Source:Eurekalert

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