Abstract
Abstract Two types of metabolic disorders are treated with transplantation: those associated with severe liver damage, like alpha‐1 antitrypsin deficiency, progressive familial cholestatic syndromes, tyrosinaemia, glycogen storage diseases, or cystic fibrosis; and those in which the liver is structurally normal, but is genetically unable to produce an essential protein, usually an enzyme, with consequent lethal systemic disease, like Crigler‐Najjar syndrome, familial hypercholesterolaemia, propionic acidaemia, or urea cycle defects. The first group of diseases, among which the most common is alpha‐1 antitrypsin deficiency, are treated by substitution of the whole liver with the donor liver, while the second group can be treated by auxiliary transplantation, to provide sufficient production of the deficient protein by the donor liver segment, while the native liver provides a safety net should the transplant fail and remains available for possible future gene therapy. In recent years, attempts have been made to treat these conditions by isolated human hepatocyte transplantation, with temporary success.
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