Emerging therapies in hepatology: liver-directed gene transfer and hepatocyte transplantation

Abstract

Gene transfer and epithelial cell transplantation technologies play an important role in the development of new therapeutic concepts for liver diseases. Although liver organ transplantation has revolutionized the treatment of a wide spectrum of acute and chronic liver diseases, gene- and cell-based therapies are emerging at an astonishing pace, because they promise to be less invasive, less costly and at least as effective as currently established therapy protocols. Experimental gene therapy models have been developed for a wide spectrum of liver diseases, including hereditary liver disorders, malignant liver disease and viral hepatitis. Hepatocyte transplantation (HcTx) is being explored as treatment of severe chronic and acute liver failure as well as for hereditary liver diseases. Most of these procedures and techniques are still experimental or have been applied to a small number of patients only. Rigorous clinical evaluation will finally demonstrate the usefulness of each new procedure in the daily clinical care of patients with liver disease. In this review, we have attempted to provide an introduction and survey of the topics of gene therapy and HcTx with specific examples of laboratory and clinical achievements highlighting potential applications in liver diseases.