Cell therapy to remove excess copper in Wilson's disease

Research output: Contribution to journalArticle

19 Citations (Scopus)

Abstract

To achieve permanent correction of Wilson's disease by a cell therapy approach, replacement of diseased hepatocytes with healthy hepatocytes is desirable. There is a physiological requirement for hepatic ATP7B-dependent copper (Cu) transport in bile, which is deficient in Wilson's disease, producing progressive Cu accumulation in the liver or brain with organ damage. The ability to repopulate the liver with healthy hepatocytes raises the possibility of cell therapy in Wilson's disease. Therapeutic principles included reconstitution of bile canalicular network as well as proliferation in transplanted hepatocytes, despite toxic amounts of Cu in the liver. Nonetheless, cell therapy studies in animal models elicited major differences in the mechanisms driving liver repopulation with transplanted hepatocytes in Wilson's disease versus nondiseased settings. Recently, noninvasive imaging was developed to demonstrate Cu removal from the liver, including after cell therapy in Wilson's disease. Such developments will help advance cell/gene therapy approaches, particularly by offering roadmaps for clinical trials in people with Wilson's disease.

Original languageEnglish (US)
Pages (from-to)70-80
Number of pages11
JournalAnnals of the New York Academy of Sciences
Volume1315
Issue number1
DOIs
StatePublished - 2014

Fingerprint

Hepatolenticular Degeneration
Cell- and Tissue-Based Therapy
Copper
Liver
Hepatocytes
Bile
Gene therapy
Poisons
Genetic Therapy
Therapy
Excess
Cells
Brain
Animals
Animal Models
Clinical Trials
Imaging techniques

Keywords

  • Bile
  • Copper
  • Excretion
  • Liver
  • Stem cell
  • Transplantation

ASJC Scopus subject areas

  • Biochemistry, Genetics and Molecular Biology(all)
  • History and Philosophy of Science

Cite this

Cell therapy to remove excess copper in Wilson's disease. / Gupta, Sanjeev.

In: Annals of the New York Academy of Sciences, Vol. 1315, No. 1, 2014, p. 70-80.

Research output: Contribution to journalArticle

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