Lentiviral Gene Transfer is a Novel Strategy in Preventing Liver Fibrosis in LEC Rats Modeling Wilson Disease

Background & Aims: To establish an efficient gene therapy in Wilson disease, the Long-Evans Cinnamon (LEC) rat model was used. We hypothesized that lentiviral gene therapy would improve liver disease and copper excretion in Wilson disease. Cell therapy was compared to systemic gene transfer for their ability to prevent liver cirrhosis. Methods: LEC rats with and without partial hepatectomy were treated either by systemic delivery of lentiviral vectors or by intrasplenic transplantation of transduced LEC-rat hepatocytes. The magnitude of successful gene transfer was assessed by RT-PCR and immunofluorescence analy…