Gene Therapy May Reverse Hemophilia

In a recent study, researchers altered the DNA of a common virus so that it would instruct the bodies of hemophiliacs to make the Factor IX (FIX) protein, which helps blood clot. They then injected six men with hemophilia B, the less-common form, with the altered virus. After a single treatment, four of the six men were successfully making the FIX protein and were able to stop weekly factor infusions. The two others have been able to stretch the time between their infusions from days to up to two weeks. To date, the procedure works to make the FIX protein in only hemophilia B patients. But, researchers say this approach could work for people who have the more common form, hemophilia A. They just need to find the right virus to deliver the genes that would help that disease. The study was published in The New England Journal of Medicine.