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April 11, 1986

Stumbling blocks pave path to clinical trials for gene therapy

JAMA. 1986;255(14):1825-1832. doi:10.1001/jama.1986.03370140019002

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"The A he goal is to alleviate human disease—and to do it with a power that has heretofore been reserved for the gods."

This statement describing gene therapy is from the introduction to an award-winning series of articles published in March in the Chicago Tribune. The introductory installment also depicts researchers "gearing up to undertake something that would have seemed like science fiction less than five years ago. They hope to break into a patient's cells and replace a malfunctioning gene."

The article reflects two popular—if not prevelent— conceptions about gene therapy: that its effect on the population will be tantamount to that of splitting the atom; and that its realization is just around the corner.

These notions need to be revised, according to panelists at the recent Symposium on Gene Therapy sponsored by the Scientists' Institute for Public Information and Stanford University. The panelists, pioneers of gene therapy, explained