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There are two treatments for extremely rare conditions approved for use in Europe; none have received FDA approval in the United States. It starts with a virus, but bioengineers remove the DNA inside, leaving the shell.This hemophilia B gene therapy uses a viral vector. The shell is filled with a FIX gene and a component that directs the vector to the liver.He felt sore afterwards, the usual aches and pains that come from hard labor, but otherwise fine.“Last year if I did that, I would be down for a whole week with joint pain,” said Hallock, 23.He has hemophilia B, a genetic disorder that impairs blood clotting.They used a FIX gene with a unique property.“This variant has five to ten times more activity than the wild-type [normal] FIX,” Sullivan said.“This means patients can receive a smaller dose of vector, which decreases the likelihood of an immune response.”To date, no trial participants have had an immune response.“This therapy looks like the leading candidate for hemophilia B.”Sullivan presented early data from four subjects, Hallock included, at the European Hematology Association's 21All four patients have severe or moderately severe hemophilia B.
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After a one-time treatment, the group maintained an average 30 percent of normal FIX several weeks later.
That's enough to control bleeds from most injuries.
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This spring, Ryan Hallock helped bulldoze 10 acres of land.