A University of Iowa researcher’s using a germ to cure a disease. Hemophilia is a genetic disorder that leaves the body unable to properly stop bleeding with the blood’s normal clotting process. Doctor Paul McCray tried using a virus to carry genetic material into the body to correct that disorder. “We’re not trying to change every cell in their body,” McCray says. “What we’re trying to do is use a gene-therapy approach to help their body make a correct copy of the protein that’s defective.” Hemophiliacs lack a protein called “Factor 8” that helps the blood clot normally. A bit of DNA designed to correct the body’s flawed chemistry is delivered by a domesticated virus, to get it into some cells within the body. They’ve modified a virus to deliver the “payload” — the genetic cargo — into cells in the liver, so they’ll work as factories to make and secrete the protein into the bloodstream. Knowing how a virus takes over cells of the body, scientists have tried that delivery method in the past. But McCray says the problem with the past experiments, which used the kind of virus that can give you the common cold, is that body gets over it. It’ll work short-term but then it’s gone, and in some case the body actually makes an immune response that destroys the cells sent in to perform a cure. Part of McCray’s study was making sure the curative effect wouldn’t just go away, once the body reacts to the invasion of foreign material. The goal of this study is to figure out a way to deliver the gene and have it stick around and persistently make the protein. That way a hemophiliac would be freed of the need to get regular injections of that Factor Eight clotting agent, the way they do now. He explains all they have to do is increase the body’s amount of normal protein, and even some improvement can let a patient lead a relatively normal life. Results in mice have shown the cure is more than temporary, though McCray cautions it’ll be a long time and require more study before the treatment can be tried on people.
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