Rewriting the "points to consider": the ethical impact of guidance document language

Human gene transfer research has been under way for nearly 10 years, with few clinically significant results to date; yet public and professional expectations still run high. Exaggerated expectations and misconceptions about gene transfer research are attributable in part to the terminology used to describe this research, which is misleading but, in the absence of more precise language, is commonly used. In particular, use of the term "gene therapy" to describe gene transfer research and use of the term "patient" in place of "subject" tend to cause both professionals and the public to confuse research with proven effective treatment. Investigators preparing to engage in human gene transfer research must use the NIH guidance document, "The Points to Consider in the Design and Submission of Protocols for the Transfer of Recombinant DNA Molecules into the Genome of One or More Human Subjects," written by the Recombinant DNA Advisory Committee, to prepare for the submission of a research protocol. Several corrections in the terminology employed by this guidance document could help to avoid misconceptions about gene transfer research and clarify both the promise and the limits of the research enterprise for investigators and subjects.