An Early History of Gene Transfer and Therapy

Abstract

The term “gene therapy” was coined to distinguish it from the Orwellian connotations of “human genetic engineering,” which, in turn, was derived from the term “genetic engineering.” Genetic engineering was first used at the Sixth International Congress of Genetics held in 1932 and was taken to mean “the application of genetic principles to animal and plant breeding.” Once the basics of molecular genetics and gene transfer in bacteria were established in the 1960s, gene transfer into animals and humans using either viral vectors and/or genetically modified cultured cells became inevitable. Despite the early exposition of the concept of gene therapy, progress awaited the advent of recombinant DNA technology. The lack of trustworthy techniques did not stop many researchers from attempting to transfer genes into cells in culture, animals, and humans. Viral genomes were used for the development of the first relatively efficient methods for gene transfer into mammalian cells in culture. In the late 1970s, early transfection techniques were combined with selection systems for cultured cells and recombinant DNA technology. With the development of retroviral vectors in the early 1980s, the possibility of efficient gene transfer into mammalian cells for the purpose of gene therapy became widely accepted.

Overview summary

This article outlines the conceptual and experimental beginnings of gene therapy. Once the basics of molecular genetics became apparent, the idea for gene therapy arose independently in the minds of perspicacious investigators. Concurrently experimentalists attempted gene transfer into mammalian cells in culture and animals in anticipation of recombinant DNA technology. Unreliable practices in early studies opened the experimental results and the entire approach of gene therapy to question, even though many of the basic methods and theories would eventually be proven correct. It was not until transfection techniques and selection systems for cultured cells were coupled with the ability to manipulate recombinant DNA that substantive progress was made in gene transfer.