Abstract |
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.
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Authors | Celine Filippi, Anil Dhawan |
Journal | Annals of the New York Academy of Sciences
(Ann N Y Acad Sci)
Vol. 1315
Pg. 50-5
(May 2014)
ISSN: 1749-6632 [Electronic] United States |
PMID | 24605914
(Publication Type: Journal Article, Research Support, Non-U.S. Gov't, Review)
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Copyright | © 2014 New York Academy of Sciences. |
Topics |
- Animals
- Cell Separation
(methods)
- Cell Survival
- Hepatocytes
(cytology, physiology, transplantation)
- Hepatolenticular Degeneration
(complications, pathology, therapy)
- Humans
- Immunosuppression Therapy
- Liver Failure, Acute
(etiology, pathology, therapy)
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