Use of Engineered Plant Cells May Help Induce Immune Tolerance in Hemophilia A Patients

Accidents as minor as a slip of the knife while chopping onions can turn dangerous for patients with hemophilia because they lack the necessary proteins in their blood to stem the flow from a wound. People with severe hemophilia typically receive regular injections of these proteins, called clotting factors, as a treatment for the disease. But up to 30 percent of people with the most common form, hemophilia A, develop antibodies that attack these life-saving proteins, making it difficult to prevent or treat excessive bleeding. Now, researchers from the University of Florida (UF) Health and the University of Pennsylvania have developed a way to thwart production of these antibodies by using plant cells to teach the immune system to tolerate rather than attack the clotting factors. The study was published in the September 4, 2014 issue of Blood. “The only current treatments against (antibody) formation cost $1 million and are risky for patients,” said Henry Daniell, Ph.D., interim chairman of biochemistry at the University of Pennsylvania School of Dental Medicine and a co-author on the study. “Our technique, which uses plant-based capsules, has the potential to be a cost-effective and safe alternative.” The study focused on hemophilia A, which occurs when babies are born with a particular defective gene on the X chromosome. Because girls have two X chromosomes — giving them two shots at having a working version of the gene — the disease typically only affects boys. Worldwide, one in 7,500 male babies is born with this disease. After receiving factor VIII treatments, between 20 and 30 percent of patients develop antibodies against the clotting protein. Instead of allowing the protein to do its job, the immune system responds to this foreign protein as an invader that must be attacked and eliminated.
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