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Bill Maurits has hemophilia B, which means his body doesn’t produce enough factor IX, a protein that clots blood. He’s at risk for bleeding and his joints are damaged from all the bruises.
In April, Maurits, an engineering designer, joined a study in which he was dosed with viruses packed with a correct version of the gene that codes for factor IX. On June 11 at the European Hematology Association’s meeting in Copenhagen, the Philadelphia company that ran the gene-therapy study, Spark Therapeutics, is presenting results on four patients, him included.
In all four, factor IX activity has reached about 30 percent of average. That’s enough to prevent bleeding when you get hit by a baseball or twist your ankle. It’s also been enough to let Maurits go without factor IX replacements since April. “There’s no other explanation than ‘It worked,’” says Maurits.
Sure, gene therapy has been tried before. What’s different is that Spark’s therapy so far appears to work well every time it’s attempted—a consistency that’s eluded previous efforts.
Read full, original post: Gene Therapy Is Curing Hemophilia