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Katherine High
Katherine High thought she was making progress. She and her team had managed to take the human gene that produces Factor IX, a blood-clotting substance that people with hemophilia lack, and slip it into a harmless virus called AAV. The virus played Trojan horse, carrying the gene into lab mice and dogs. There, it worked its way into the animals' DNA and produced enough clotting factor to cure their hemophilia.


But when Dr. High, a professor of pediatrics at Children's Hospital of Philadelphia, and her colleagues launched human trials in 2001, the picture darkened. They injected the gene-carrying viruses into the livers of seven patients. Two produced measurable amounts of Factor IX, 3 percent and 12 percent of the normal level. But within a few weeks the levels became undetectable, Dr. High reported last year. The patients' immune systems had apparently killed the cells containing the inserted gene.


Dr. High had some ideas about how to fix that. But in May the biotech company sponsoring her work bailed out.


So it has gone for gene therapy, the great hope of...