Prospects for Epithelial Gene Therapy

Abstract

There is considerable excitement about the possibility of somatic cell gene therapy, that is, the introduction and expression of defined genes into cells for the purpose of providing a needed gene product. Most current research on gene therapy has been focused on the use of marrow stem cells as a therapeutic vehicle (Belmont et al., 1986). Inherited hematological disorders, such as severe combined immunodeficiency caused by adenosine deaminase deficiency, are diseases that might be amenable to this form of therapy (Kellems et al., 1985). A possible approach would be through genetic transfer of a recombinant adenosine deaminase gene into autologous marrow stem cells, transplantation of the transformed cells into the patient, and establishment of a population of stem cells that give rise to immunologically competent lymphoid cells. Although many questions need to be answered before we know the efficacy of such therapy, the National Institute of Health has issued guidelines for its use, and clinical trials have already begun (Culliton, 1989).