Transgenic Sertoli Cells as a Vehicle for Gene Therapy

Abstract

Gene therapy involves the manipulation of genetic material to replace defective or deficient proteins to restore function in disease states. These genes are introduced into cells by mechanical, chemical, and biological approaches. To date, cell-based gene therapy has been hampered by the lack of an abundant, safe, and immunologically acceptable source of tissue. As an alternative, transgenic animals designed to produce therapeutic proteins could overcome some of the issues facing gene therapy but the problem of immune rejection of the tissue remains. This article reports on recently published work indicating the potential to use transgenic Sertoli cells surviving in an allogeneic host by virtue of their ability to create a locally immunoprivileged environment, thereby providing for the continued delivery of a therapeutic protein to the systemic circulation.