Hepatocyte transplantation for inborn errors of metabolism

Liver transplantation for inborn errors of metabolism has proved effective in some (mostly liver-associated) inborn errors of metabolism. Significant morbidity and mortality rates have been extensively reported due to disease recurrence or to complications of the immunosuppressive regimen. On the basis of clinical trials in animals as well as in humans, the use of isolated hepatocytes offers a unique opportunity for treating inborn errors of metabolism. The state of art of the technique applied to this field is reviewed here and related practical problems are examined. No final conclusions can be drawn, but further insight into the use of alternative sources of cells, including stem/progenitor cells associated with cryobiology and immunological research, will offer new opportunities for cell therapy for inborn errors of metabolism in the near future.