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Wilson病(WD)是一种常染色体隐性遗传性铜代谢障碍性疾病,ATP7B基因突变是该病的根本病因。基因及细胞治疗是最可能从根本治愈本病,最具前景的治疗途径。目前,国内外的基础研究主要是以质粒、腺病毒、慢病毒等为载体介导基因治疗。细胞移植的方法有肝细胞移植、胎儿肝祖细胞移植、骨髓干细胞移植、胚胎干细胞移植。本文就以上方法的基础研究以及对促进干细胞向肝细胞方向分化的因素作一综述。各种基因及细胞移植技术均能达到一定的治疗动物模型作用,但仍存在许多不足之处。促进干细胞向肝细胞方向分化的肝细胞核因子是重要的研究方向之一。
Wilson’s disease (WD) is an autosomal recessive copper metabolism disorders, ATP7B gene mutation is the underlying cause of the disease. Gene and cell therapy are the most promising ways to cure this disease from the very beginning. At present, the basic research at home and abroad mainly uses plasmid, adenovirus and lentivirus as vectors to mediate gene therapy. Cell transplantation methods include liver cell transplantation, fetal liver transplantation, bone marrow stem cell transplantation, embryonic stem cell transplantation. This article reviews the above basic research methods and the factors that promote the differentiation of stem cells into hepatocytes. A variety of gene and cell transplantation techniques can achieve a certain role in the treatment of animal models, but there are still many shortcomings. It is one of the most important research directions to promote the differentiation of stem cells into hepatocytes.