Emerging insights into liver-directed cell therapy for genetic and acquired disorders

Treatment of acute or chronic liver diseases by cell transplantation is an attractive prospect because organ shortages greatly restrict liver transplantation. Moreover, a variety of genetic deficiency states affecting extrahepatic organs are amenable to liver-directed cell therapy. While the initial clinical experience with liver cell transplantation has been encouraging, further advances in several areas are necessary to improve these results. Insights into how engraftment and proliferation of transplanted cells may be modulated to obtain therapeutically effective masses of transplanted cells will be important in this pursuit. Studies of cell therapy in animal models of specific diseases have provided insights into the development of clinically relevant strategies for various disorders. Also, identification of suitable cell types, including stem/progenitor cells that could be expanded and manipulated in cell culture conditions, has begun to provide important new information for cell therapy. Similarly, advances in cryopreservation of cells and prevention of allograft rejection offer ways to accomplish cell therapy in an effective manner. Taken together, these advances indicate that liver-directed cell therapy will be well positioned in the near future to play significant roles in transplantation medicine.