Cutaneous gene therapy.

Abstract

Gene therapy efforts in a variety of tissues have foundered on fundamental technologic barriers, such as difficulties in achieving high-efficiency gene transfer to diseased tissues and in sustaining delivered transgene production. The skin offers an attractive tissue for development of approaches to therapeutic gene delivery by virtue of its accessibility for regulation by topical agents, the ease of gene transfer into cutaneous tissues, and the ready ability to monitor the impact of somatic gene transfer. With the ability of the skin to deliver therapeutic polypeptides to the systemic circulation and the recent molecular characterization of monogenic skin diseases, efforts to target genes to the skin are expected to accelerate. The current status of gene therapy efforts is reviewed, with a special focus on the skin.