Hepatocyte transplantation for inherited metabolic diseases of the liver

Inherited metabolic diseases of the liver are characterized by deficiency of a hepatic enzyme or protein often resulting in life‐threatening disease. The remaining liver function is usually normal. For most patients, treatment consists of supportive therapy, and the only curative option is liver tra...

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Veröffentlicht in:Journal of internal medicine 2012-09, Vol.272 (3), p.201-223
Hauptverfasser: Jorns, C., Ellis, E. C., Nowak, G., Fischler, B., Nemeth, A., Strom, S. C., Ericzon, B. G.
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container_end_page 223
container_issue 3
container_start_page 201
container_title Journal of internal medicine
container_volume 272
creator Jorns, C.
Ellis, E. C.
Nowak, G.
Fischler, B.
Nemeth, A.
Strom, S. C.
Ericzon, B. G.
description Inherited metabolic diseases of the liver are characterized by deficiency of a hepatic enzyme or protein often resulting in life‐threatening disease. The remaining liver function is usually normal. For most patients, treatment consists of supportive therapy, and the only curative option is liver transplantation. Hepatocyte transplantation is a promising therapy for patients with inherited metabolic liver diseases, which offers a less invasive and fully reversible approach. Procedure‐related complications are rare. Here, we review the experience of hepatocyte transplantation for metabolic liver diseases and discuss the major obstacles that need to be overcome to establish hepatocyte transplantation as a reliable treatment option in the clinic.
doi_str_mv 10.1111/j.1365-2796.2012.02574.x
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subjects Adaptive Immunity
Animals
Biological and medical sciences
Cell Culture Techniques
Cell Transplantation - methods
Cellular Senescence
Cryopreservation
experimental
General aspects
Hepatocytes - cytology
hepatology
Humans
Immunity, Innate
Immunosuppression
liver
Liver Diseases - therapy
Medical sciences
Metabolism, Inborn Errors - therapy
surgery
Tissue Donors
Transplantation Conditioning
transplantation immunology
α-1-antitrypsin deficiency
title Hepatocyte transplantation for inherited metabolic diseases of the liver
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