Current status of gene therapy in gastroenterology

The potential role of genetic intervention extends from diseases caused by single gene defects, through severe viral infections, to polygenic disorders, such as diabetes mellitus and arteriosclerosis. However, gene therapy can be defined as the introduction and expression of an exogenous gene in human cells for therapeutic benefit, and is conventionally restricted to human diseases associated with single gene defects. There are wider opportunities for genetic intervention and these include strategies to reduce or block gene expression as well as the introduction of nonmammalian genes. The rapid progress in our understanding of some of the molecular mechanisms involved in the pathogenesis of cancer and metabolic disorders, coupled with the development of gene delivery vector technology, has urged us to consider novel genetic approaches to digestive diseases. There is no shortage of ideas and applications for genetic intervention in human diseases, but there are great limitations not only with the efficiency and targeting of the present generation of gene transfer vectors but also with our incomplete understanding of transcription control.