The Potential of Gene Therapy in the Peritoneal Cavity

Abstract

Gene therapy is a promising new treatment modality based on molecular genetic modification to achieve a therapeutic benefit. We believe that gene therapy in the peritoneal cavity holds considerable promise, and we describe strategies by which genetic modification can be used to treat a variety of disease states or conditions. First, we can envision a strategy, based on genetic modification of the peritoneal membrane, to improve the practice of peritoneal dialysis through the production of proteins that would be of therapeutic value in preventing membrane damage and in preserving or enhancing its function as a dialyzing membrane. Second, the membrane could be genetically modified for either local or systemic delivery of therapeutic proteins. This approach could be applied to a variety of pathologies or conditions that require either sustained or transient delivery of therapeutic proteins, such as enzymes or growth factors. Third, gene transfer has already been incorporated into several strategies for the treatment of intra-abdominal carcinomas, and it has been effective in animal models of ovarian and bladder cancer and of peritoneal mesothelioma. Finally, gene transfer can be a valuable tool in increasing our understanding of the biology of the peritoneal membrane. By being able to manipulate the expression of specific genes through gene transfer, their role in various (patho)physiological processes can be identified. In summary, gene therapy in the peritoneal cavity has significant potential to address a variety of diseases or pathophysiological conditions, and to further our knowledge of peritoneal cavity biology.