Inherited metabolic liver disease

Curr Opin Gastroenterol. 2005 May;21(3):275-82. doi: 10.1097/01.mog.0000159821.78532.21.

Abstract

Purpose of review: The purpose of this review is to identify and discuss recent findings related to inherited metabolic disorders of the liver that increase our understanding of the pathophysiology and treatment for hemochromatosis and other iron overload disorders, Wilson disease and alpha one antitrypsin deficiency.

Recent findings: The main theme in the recent discoveries for both iron overload disorders and Wilson disease is our increasing understanding that the phenotypic expression of these disorders are greatly influenced by genes involved in the metabolic pathways for these metals, or influence the progression of liver disease independent of metal metabolism. For example, the role of hepcidin dysregulation in hemochromatosis has been a surprising discovery that provides some mechanistic understanding for the increased iron absorption that is present in this disorder.

Summary: Given the recent explosion of information on iron and copper metabolism and the cellular processing of alpha one antitrypsin, the highlights reviewed in this article will help the reader keep up to date with the current understanding of these diseases and potential future approaches to their treatment.

Publication types

  • Review

MeSH terms

  • Hemochromatosis / genetics
  • Hemochromatosis / physiopathology*
  • Hemochromatosis / therapy*
  • Hepatolenticular Degeneration / genetics
  • Hepatolenticular Degeneration / physiopathology*
  • Hepatolenticular Degeneration / therapy*
  • Humans
  • alpha 1-Antitrypsin Deficiency / genetics
  • alpha 1-Antitrypsin Deficiency / physiopathology*
  • alpha 1-Antitrypsin Deficiency / therapy*