Hemophilia is an ideal model for gene therapy, speaker after speaker repeated during the American Society for Gene Therapy's (ASGT) June meeting in Washington, D.C. Successfully providing a single protein a few times should, in theory, cure the disease, they stated. And once introduced via vector, a therapeutic clotting protein need not be continuously produced or regulated, they added. What they did not mention--at least not as openly--was the double-edged nature of that "ideal" status. In other words, if gene therapy doesn't work in hemophilia models, in what disease model will it work?

"The stakes are incredibly high," acknowledged James Wilson, ASGT president and director of the Institute for Human Gene Therapy at the University of Pennsylvania in Philadelphia, at the end of a session focusing on treating the disease. Within a year, scientists may find out if the stakes are too high. Phase I clinical trials using a...

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