Cell therapy to remove excess copper in Wilson's disease.
Cell therapy to remove excess copper in Wilson's disease.
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DOI：
10.1111/nyas.12450
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发表时间：
2014-05
影响因子：
5.2
通讯作者：
Gupta S
中科院分区：
文献类型：
--
作者：
Gupta S
为了通过细胞疗法实现肝豆状核变性的永久矫正，最需要的是替换健康的肝细胞。肝内胆汁中atp7b依赖性铜转运存在生理需要，威尔逊氏病缺乏这一生理需要，导致铜在肝脏或脑内进行性积累，并伴有器官损伤。用健康肝细胞重新填充肝脏的能力增加了威尔逊氏病细胞治疗的可能性。治疗原则包括重建胆管网络以及移植肝细胞的增殖，尽管肝脏中含有有毒的铜。尽管如此，在动物模型中进行的细胞治疗研究表明，在肝豆状核变性患者与非病变患者中，移植肝细胞驱动肝脏再生的机制存在重大差异。最近，无创成像技术被用于证明肝内铜的去除，包括在威尔逊氏病的细胞治疗后。这些进展将有助于推进细胞/基因治疗方法，特别是通过为威尔森氏症患者的临床试验提供路线图。
To achieve permanent correction of Wilson’s disease by a cell therapy approach, replacement of healthy hepatocytes will be most desirable. There is a physiological need for hepatic ATP7B-dependent copper transport in bile, which is deficient in Wilson’s disease, producing progressive copper accumulation in the liver or brain with organ damage. The ability to repopulate the liver with healthy hepatocytes raised possibilities for cell therapy in Wilson’s disease. Therapeutic principles included reconstitution of bile canalicular network as well as proliferation in transplanted hepatocytes, despite toxic amounts of copper in the liver. Nonetheless, cell therapy studies in animal models elicited major differences in the mechanisms driving liver repopulation with transplanted hepatocytes in Wilson’s disease versus nondiseased settings. Recently, noninvasive imaging was developed to demonstrate copper removal from the liver, including after cell therapy in Wilson’s disease. Such developments will help advance cell/gene therapy approaches, particularly by offering roadmaps for clinical trials in people with Wilson’s disease.
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