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Horrible Death of US Woman Raises Questions on Gene Therapy
September 17, 2007 at 11:54 AM
General Health News
The 36-year-old Mohr so swollen by internal bleeding and her failing kidneys that her husband decided against bringing their 5-year-old daughter to say goodbye. The girl wouldn't have recognized her mother.
The nationwide experiment in which Mohr was a volunteer has since been halted. Targeted Genetics Corporation of Seattle, the sponsors say no other problems have been reported, and the company believes patients were adequately informed of the treatment's risks.
U.S. health officials are investigating Mohr's death, and the case is expected to be discussed Monday by advisers to the National Institutes of Health (NIH).
There have been more than 800 gene therapy studies involving 5,000 U.S. patients since the NIH approved the nation's first human gene transfer study in 1989. Yet there are no approved therapies despite 17 years of research, and the only major success — a cure for the rare inherited immune disorder known as "bubble boy disease" — came with a high cost: leukemia linked in 2003 to the virus that delivered the treatment.
Still, the 1999 death of Arizona teenager Jesse Gelsinger is the only reported fatality definitively linked with a U.S. gene therapy study, an NIH spokesman said. And Dr. Theodore Friedmann, who once headed the NIH committee that oversees gene therapy experiments, said developments in medicine often come with problems, even death.
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