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...
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