Scientific aspects of gene therapy in melanoma

Abstract

Melanoma is one of the first tumors in which gene therapy protocols are being tested. The promising results of in vitro and animal studies are now being translated into phase I studies in patients with metastatic disease. Attention is being paid to the safety of the various techniques for gene transfer. As yet, almost all protocols involve ex vivo delivery of genetic material because we lack techniques to ensure 100% transduction of target cells in vivo. The majority of studies in animal models and most current clinical trials involve cytokine gene-modified cells. For melanoma, a number of the target epitopes for cytotoxic lymphocytes have been discovered so that rational testing of the immunomodulatory effects of such therapy is now possible.