Current status of human hepatocyte transplantation and its potential for Wilson's disease

Ann N Y Acad Sci. 2014 May;1315:50-5. doi: 10.1111/nyas.12386. Epub 2014 Mar 7.


Wilson's disease (WD) is a genetic disorder of liver copper excretion leading to its accumulation in various vital organs like the liver, brain, and kidneys. Drugs such as penicillamine, trientine, and zinc salts are the mainstay of treatment, with good outcomes; but nonresponders or a lack of compliance to the drug treatment can result in disease progression and acute liver failure (ALF). Current treatment for WD with ALF is an emergency liver transplantation and lifelong immunosuppression. Human hepatocyte transplantation (HTx) is increasingly used as treatment for liver-based metabolic defects. HTx may benefit WD patients with ALF, either as transient support until chelation treatment shows its effect or as a definitive cure through liver repopulation by healthy donor cells, as shown in animal models of WD. Although clinical trials of HTx have already proven safety and efficacy in different ALF etiologies, it remains to be demonstrated similarly in cases of WD.

Keywords: Wilson's disease; acute liver failure; hepatocyte; transplantation.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Cell Separation / methods
  • Cell Survival
  • Hepatocytes / cytology
  • Hepatocytes / physiology
  • Hepatocytes / transplantation*
  • Hepatolenticular Degeneration / complications
  • Hepatolenticular Degeneration / pathology
  • Hepatolenticular Degeneration / therapy*
  • Humans
  • Immunosuppression Therapy
  • Liver Failure, Acute / etiology
  • Liver Failure, Acute / pathology
  • Liver Failure, Acute / therapy