24 Human Gene Therapy: Public Policy and Regulatory Issues

Abstract

Although human gene therapy owes its current status to the development of recombinant DNA technology, several prescient investigators postulated that gene transfer into cells might be possible, even before all of the basic experiments in molecular genetics had been performed (Wolff and Lederberg 1994). In the 1960s, Edward Tatum suggested that viruses could effectively be used for man’s benefit by using them to introduce genes into cells, and he actually described a set of conditions that accurately characterizes ex vivo transduction of target cells (Tatum 1966). Joshua Lederberg anticipated that the in vitro culture of germ cells would be possible and that it would be possible to interchange chromosomes and their segments. Furthermore, he predicted that ultimately it might be possible to directly control nucleotide sequences in human chromosomes along with recognition, selection, and integration of the desired genes (Lederberg 1968). After announcing the first successful in vitro synthesis of DNA, Arthur Kornberg predicted that it might be possible to attach a gene to a harmless viral DNA and to use such a virus to cure a patient suffering from a hereditary defect by delivering a gene to the cells (Kornberg 1971). In 1968, one of the authors (W.F.A.) submitted an article to the New England Journal of Medicine in which he described how human gene therapy could be accomplished. Although the article was not accepted because it was too speculative, a portion of the text is instructive as it stated that, “In order to insert a correct gene into...