A cut can be life-threatening for people with hemophilia, whose bodies dont produce enough of a protein that prevents prolonged bleeding.
Now University of Florida researchers may be one step closer to finding a safe way to spur production of this missing protein in patients with the most common form of the hereditary bleeding disorder. Using a dormant strand of DNA that has quietly existed in fish for millions of years, the researchers replaced the faulty gene responsible for the disease in neonatal mice, according to findings published online this month in the journal Molecular Therapy.
"The degree to which these patients have problems from hemophilia stems from how much of this protein, factor VIII, is missing," said Bradley Fletcher, M.D., Ph.D., a UF assistant professor of pharmacology and one of the lead authors of the study. "If they have very low levels of it, they have lifelong problems of bleeding, but whats even more problematic for them is they bleed into their joints, knees, hips and ankles, which limits their mobility."
April Frawley Birdwell | EurekAlert!
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