Gene Therapy

Gene therapy has provided an opportunity to supply genetic material to individuals and possibly alleviate certain diseases. This chapter examines the different methods of gene transfer and various conditions under which they have been used. The properties of retroviruses make them a natural choice as vectors for the transmission and expression of foreign genes. Retroviruses predominantly infect vertebrates and most have a similar structure, mechanism of replication, and genome organization. Cytokines have also been transferred to tumor-infiltrating lymphocytes. Vectors derived from herpes simplex-1 have been able to deliver transgenes to postmitotic neurons. Nucleic acid inoculation in vivo has been utilized as a research tool for a number of years. Many of the animal models currently being used in gene therapy were bred for a given condition, such as the Watanabe rabbit, which has extremely high cholesterol. The National Institutes of Health’s Recombinant DNA Advisory Committee was established to consider the science and ethics of gene therapy.