caa a22 4500 467933197 CHVBK 20180406152949.0 cr unu---uuuuu 170328e20060401xx s 000 0 eng 10.1007/s10545-006-0245-8 doi (NATIONALLICENCE)springer-10.1007/s10545-006-0245-8 Hepatocyte transplantation for liver-based metabolic disorders [Elektronische Daten] [Anil Dhawan, Ragai Mitry, Robin Hughes] Summary: Hepatocyte transplantation is being investigated as an alternative to orthotopic liver transplantation in patients with liver-based metabolic disorders. The progress made in this field to date is reviewed. Protocols have been developed using collagenase perfusion to isolate human hepatocytes from unused donor liver tissue. Hepatocytes with a high viability can often be obtained and can be cryopreserved for later use, though with loss of function on thawing. For clinical use, hepatocytes must be prepared in clean GMP conditions with cells meeting criteria of function and lack of microbial contamination before patient use. Hepatocytes are infused intraportally into the patient's liver, where a proportion of cells will engraft and replace the deficient metabolic function without the need for major surgery. Twenty patients have now received hepatocyte transplantation, including eight children at King's College Hospital. There was a range of aetiologies of liver disease: familial hypercholesterolaemia, Crigler-Najjar syndrome type 1, urea cycle defects, infantile Refsum disease, glycogen storage disease type Ia, inherited factor VII deficiency and progressive familial intrahepatic cholestasis type 2. Clinical improvement and partial correction of the metabolic abnormality was observed in most cases. Considerable progress has been made in developing the technique, but hepatocyte transplantation is limited by the available supply of liver tissue. Hepatocytes derived from stem cells could provide alternative sources of cells in the future. SSIEM and Springer, 2006 Dhawan Anil Paediatric Liver Service, King's College Hospital, Denmark Hill, SE5 9RS, London, UK aut Mitry Ragai Institute of Liver Studies, King's College London School of Medicine, King's College Hospital, London, UK aut Hughes Robin Institute of Liver Studies, King's College London School of Medicine, King's College Hospital, London, UK aut Journal of Inherited Metabolic Disease Kluwer Academic Publishers 29/2-3(2006-04-01), 431-435 0141-8955 29:2-3<431 2006 29 10545 https://doi.org/10.1007/s10545-006-0245-8 text/html Onlinezugriff via DOI 1 research-article jats NATIONALLICENCE 856 40 https://doi.org/10.1007/s10545-006-0245-8 text/html Onlinezugriff via DOI NATIONALLICENCE 700 1- Dhawan Anil Paediatric Liver Service, King's College Hospital, Denmark Hill, SE5 9RS, London, UK aut NATIONALLICENCE 700 1- Mitry Ragai Institute of Liver Studies, King's College London School of Medicine, King's College Hospital, London, UK aut NATIONALLICENCE 700 1- Hughes Robin Institute of Liver Studies, King's College London School of Medicine, King's College Hospital, London, UK aut NATIONALLICENCE 773 0- Journal of Inherited Metabolic Disease Kluwer Academic Publishers 29/2-3(2006-04-01), 431-435 0141-8955 29:2-3<431 2006 29 10545 Metadata rights reserved Springer special CC-BY-NC licence nationallicence BK010053 XK010053 XK010000 NATIONALLICENCE NATIONALLICENCE NL-springer