ATP7B gene therapy of autologous reprogrammed hepatocytes alleviates copper accumulation in a mouse model of Wilson’s disease,Hepatology

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ATP7B gene therapy of autologous reprogrammed hepatocytes alleviates copper accumulation in a mouse model of Wilson’s disease
Hepatology ( IF 12.9 ) Pub Date : 2022-03-27 , DOI: 10.1002/hep.32484
Hong‐Xia Cai ₁ , Cheng Xin ₂ , Xiao‐Ping Wang ₁

Affiliation

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 autologous reprogrammed, ATP7B gene-restored hepatocytes using a mouse model of WD.

中文翻译：

自体重编程肝细胞的 ATP7B 基因治疗可减轻威尔逊病小鼠模型中铜的积累

威尔逊病 (WD) 是一种罕见的遗传性疾病，由于ATP7B基因突变，导致病理性铜储存主要在肝脏和神经系统中。肝细胞移植显示出治疗潜力；然而，这种策略经常受到优质供体细胞短缺和同种异体免疫排斥的阻碍。在这项研究中，我们旨在使用 WD 小鼠模型评估自体重编程、 ATP7B基因恢复的肝细胞的功能和功效。

更新日期：2022-03-27

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