Intrahepatic transplantation of normal hepatocytes prevents Wilson's disease in long-Evans cinnamon rats

Background & Aims: Long-Evans cinnamon (LEG) rats are an animal model of Wilson's disease, This study investigated whether intrahepatic transplantation of normal hepatocytes can prevent Wilson's disease in LEC rats, Methods: Hepatocytes of newborn Long-Evans agouti (LEA) rats were transplanted into the livers of LEC rats before onset ni hepatic disease; Expression of normal transcripts from the gene linked to Wilson's disease was investigated at 30 weeks of age by re. verse-transcription polymerase chain reaction and in situ hybridization. Results: Eight of 16 (50.0%) untreated LEC rats died of hepatic failure during 20-30 weeks of age, Of the 27 LEC vats with transplanted cells, 2 (7.4%) died of hepatic failure and 4 died of ileus complicated by the surgical treatment, In the recipient livers, the transplanted cells comprised 4%-20% of the hepatocyte populations, expressing normal messenger RNA transcribed from the Wilson's disease gene, and hepatic copper deposition was reduced to approximately 60% of that in untreated LEG rats, Conclusions: Transplantation of normal hepatocytes prevents Wilson's disease in LEC rats.