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Unformatted text preview: Ask the Experts- May 13, 2008 How does gene therapy work? Arthur Nienhuis, a hematologist at St. Jude Children's Research Hospital in Memphis, Tenn., and president of the American Society of Gene Therapy, responds: Gene therapy is the addition of new genes to a patient's cells to replace missing or malfunctioning genes. Researchers typically do this using a virus to carry the genetic cargo into cells, because thats what viruses evolved to do with their own genetic material. The treatment, which was first tested in humans in 1990, can be performed inside or outside of the body. When its done inside the body, doctors may inject the virus carrying the gene in question directly into the part of the body that has defective cells. This is useful when only certain populations of cells need to be fixed. For example, researchers are using it to try to treat Parkinson's disease, because only part of the brain must be targeted. This approach is also being used to treat eye diseases and hemophilia, an inherited disease that leads to a high risk for...
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- Summer '08