Manipulation of Biliary Lipids by Gene Therapy: Potential Consequences for Patients with Progressive Familial Intrahepatic Cholestasis

Ronald   P.J.   Oude Elferink

Abstract

Gene therapy constitutes a great promise for the treatment of inherited diseases as well as cancer. Although the principle is extremely elegant, reality proves that several important problems remain to be solved before gene therapy becomes a standard application for these conditions. Meanwhile, and because of these problems alternatives are being considered as well. For the treatment of hepatic inherited disorders, hepatocyte transplantation has proven to be an attractive alternative, although this form of therapy also remains experimental at this moment. Problems and possibilities are discussed with the inherited disease, Progressive Familial Intrahepatic Cholestasis, as an example.

Keywords: somatic gene therapy, adeno associated virus (aav), lentiviral vectors, hepatocytes, transplantation, liver damage, jaundice, fic gene, pfic type