Paediatric patients with life-threatening liver-based metabolic disorders require organ transplantation even though their metabolic diseases are typically the result of a single enzyme deficiency, and the liver otherwise functions normally. These disorders include Crigler–Najjar syndrome type 1, familial hypercholesterolemia, factor VII deficiency, glycogen storage disease type I, infantile Refsum’s disease, progressive familial intrahepatic cholestasis (PFIC) type 2, and a variety of urea cycle defects.
Hepatocyte transplantation (HT) has been employed as an alternative to whole organ transplantation to replace the enzyme deficiency typical of these disorders. However, while HT can be employed safely in humans, its applicability remains limited by technical issues and particularly a relatively poor initial and long-term hepatocyte engraftment that limits successful treatment of liver based metabolic deficiencies. The use of implantable liver tissue will resolve many of the problems typical of the current methodology for HT and will ensure a prolonged therapeutic effect