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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
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
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。