Gene therapy: applications to the treatment of gastrointestinal and liver diseases

Abstract

There has been much progress in our understanding of molecular mechanisms in the pathogenesis of inherited metabolic disorders. In addition, powerful new molecular techniques have made possible phenotypic alterations by delivery of foreign genes to target cells. As a result, concepts and methods that would have been considered purely science fiction 10 years ago can now be found in human clinical trials engaged in the treatment of these disorders. In this review, we have attempted to provide an introduction and survey of the topic of gene therapy, with specific examples of laboratory and clinical achievements to date, and highlights on potentials for applications in digestive diseases.