유전성 대사 질환 동물 모델에서의 줄기 세포 치료

Stem cell therapy in animal models of inherited metabolic diseases

Orthotopic liver transplantation is the treatment of choice for inherited metabolic diseases. However, the supply of donor organs is limiting and therefore many patients cannot benefit from this therapy. In contrast, hepatocytes can be isolated from a single donor liver. They can be transplanted into several recipients, and this procedure may help overcome the shortage of donor livers. A great deal of work with animal models indicates that hepatocytes transplanted into the liver or spleen can survive, function, and participate in the normal regenerative process. Recent clinical studies suggest that hepatocyte transplantation may be useful for bridging patients to whole organ transplantation and for providing metabolic support during liver failure and for replacing whole organ transplantation in certain inherited metabolic diseases. Nowadays, hepatocytes from various stem cells have been regarded as an another cell source for treatment of inherited metabolic diseases. Although cell therapy using stem cells for inherited metabolic disease patient has been accepted only as an experimental trial yet, hepatocytes from stem cells can solve a lot of obstacles in the treatment of inherited metabolic diseases.