Article
ATP7B gene therapy of autologous reprogrammed hepatocytes alleviates copper accumulation in a mouse model of Wilson's disease.
Hepatology (Baltimore, Md.) - 1 Oct 2022
Cai Hongxia, Cheng Xing, Wang Xiao-Ping
Abstract excerpt
BACKGROUND AND AIMS: Wilson's disease (WD) is a rare hereditary disorder due to ATP7B gene mutation, causing pathologic copper storage mainly in the liver and neurological systems. Hepatocyte transplantation showed therapeutic potential; however, this strategy is often hindered by a shortage of quality donor cells and by allogeneic immune rejection. In this study, we aimed to evaluate the function and efficacy of...
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