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A Prescription for Gene Therapy
The First Cabo Gene Therapy Working Group
Introduction by
Barbara J. Culliton

In the Literature
Gene therapy is in better shape than many people think, according to a group of researchers and others who got together in Cabo San Lucas, Mexico, to review the state of their field. The meeting symposium marked the anniversary of ten years of human studies of gene therapy. Physicians, researchers from the biotechnology industry, and bioethicists were among those who gave the field a surprisingly favorable report. In essence they said that, despite some much-publicized setbacks, the real question to ask is not, "Why has gene therapy failed to cure disease," but "How has it done so well overall in such a short time." After all, drug development routinely takes a decade or even more. So there is no reason to write gene therapy off yet.

The report concludes with this summary: "It is a difficult time for gene therapy, but not a fatal one. In many ways, we are experiencing the growing pains of any young field of medicine. Granted, these are magnified for gene therapy, given the uneasy public reaction to the notion of genetic intervention and the intense media scrutiny following recent events. However, just as we have the opportunity to push this young field ahead, we also have the responsibility to adhere to current regulations and to follow tried and true models for developing what are, in essence, new drugs. Far from being a time to retreat, we need to stand up to the public and state clearly our successes and shortcomings, capitalizing on the first and working to change the second."

The full text of "A Prescription for Gene Therapy," from the May issue of Molecular Therapy, is reprinted here.

--Barbara J. Culliton

Download A Prescription for Gene Therapy (pdf)

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