254 GENE THERAPY FOR CONGENITAL HYPERBILIRUBINEMIA

Most of the congenital errors of liver metabolism affect one single pathway and spare the general function and structure of the liver. Gene transfer into hepatocytes might be an alternative treatment of many inborn errors of the liver metabolism. We study gene therapy of the congenital unconjugated hyperbilirubinemia (Crigler-Najjar type 1) in a rat model (Gunn-rat). The gene for UDPGT has been cloned. We have constructed an expression vector containing the B-UGT-1 cDNA. We are busy to characterize the expression of the B-UDPGT cDNA by western-blotting and in a functional assay for bilirubin glucuronidation in microsomes prior to the use for gene transfer into hepatocytes in vitro and in vivo.