Study holds hope for next generation hemophilia treatment
Researchers have doubled the potency of a protein that drives blood to clot, according to research to be published in the July 26 edition of Biochemistry. The study results may 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 factor VIII, one of several proteins that enable 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 of factor VIII for 20 years, and are making subtle changes in the protein with the goal of offering more effective, less burdensome treatment.
Greg Williams | EurekAlert!
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