In situ retrovirus-mediated gene transfer into the liver

Gene transfer into hepatocytes is a promising approach for the treatment of genetic liver diseases. Candidate diseases for human trials are life-threatening disorders resulting from a single genetic defect that do not compromise other liver functions and the organization of the hepatic tissue. Orthotopic liver transplantation has been successfully performed in patients with genetic liver disease, but then use is limited by the high mortality risk, the need for chronic immunosuppression, and the availability of organs. Gene therapy would pro vide an alternative to transplantation by restoring the expression of the defective gene in an organ that is otherwise structurally and functionally normal.