Demonstrating Potential of Cell Therapy for Wilson's Disease with the Long-Evans Cinnamon Rat Model.

doi:10.1007/978-1-4939-6506-9_11

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Article

Demonstrating Potential of Cell Therapy for Wilson's Disease with the Long-Evans Cinnamon Rat Model.

Methods in molecular biology (Clifton, N.J.) - 1 Jan 2017

Jaber Fadi Luc, Sharma Yogeshwar, Gupta Sanjeev

Abstract excerpt

Wilson's disease (WD) is characterized by the inability to excrete copper (Cu) from the body with progressive tissue injury, especially in liver and brain. The molecular defect in WD concerns mutations in ATP7B gene leading to loss of Cu transport from the hepatocyte to the bile canaliculus. While drugs, e.g., Cu chelators, have been available for several decades, these must be taken lifelong, which can be...

Topics

Animals
Cell Transplantation
Copper
Copper-Transporting ATPases
Disease Models, Animal
Genetic Therapy
Hepatobiliary Elimination
Hepatocytes
Hepatolenticular Degeneration
Humans
Liver
Liver Transplantation
Mutation
Rats
Rats, Inbred LEC

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PMID: 27830552
DOI: 10.1007/978-1-4939-6506-9_11
PMCID: PMC5458614

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