Somatic Gene Therapy

Abstract

The initial social and political barriers preventing the use of gene therapy in humans have essentially been overcome by a variety of committees, and by both the NIH and the FDA. During the past year, at least two experiments have been approved for somatic gene therapy. In principle, gene therapy does not differ from any other form of therapy; e.g., giving a drug to a cancer patient or an antibiotic to a patient with infection. The requirements for ethical and scientifically valid gene therapy experiments, as in all other clinical research, involve an estimation of the risk:benefit ratio. If the risk is small and the benefit potentially great, then the experiment should be approved; if the risk is great and potential benefits small, the experiment should not be approved. In addition, if there is no appropriate way to evaluate the effects of an experiment, it should not be performed.