ABSTRACT
Gene therapy, the transfer of genes into cells for a therapeutic effect, is an experimental approach to the treatment of disease. The first clinically applicable system for efficiently delivering genes into mammalian cells was developed in the early 1980s and was based on a genetically engineered retrovirus, which, as part of its lifecycle, stably integrates its genome into the target cell’s chromosomal DNA. Using recombinant DNA technology perfected in the mid-1970s, investigators replaced the viral genes with therapeutic genes and the resulting recombinant retrovirus shuttled these genes into the target cells. The potential applications of gene therapy are far reaching (Table 18.1) since there are over 4000 known human genetic diseases (many of which have no viable treatment) and virtually every human disease is profoundly influenced by genetic factors [1]. In addition to inherited diseases, other viable targets for gene therapy include more prevalent disorders that show a complex genetic dependence (i.e., cancer and heart disease) as well as infectious diseases (i.e., human immunodeficiency virus) and applications in tissue engineering [1,2].
