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Wilson病（WD）是一种ATP7B基因突变导致铜排泄障碍的常染色体隐性遗传性单基因疾病。药物治疗是目前WD的主要治疗方法，药物应答不佳或急性肝衰竭需考虑肝移植，但面临药物治疗依从性以及不良反应、肝源的紧缺等问题。WD的基因治疗有可能永久纠正异常铜代谢，是目前研究的焦点问题。现围绕基因治疗的载体和CRISPR/Cas9基因编辑系统总结WD基因治疗的研究进展。“,”Wilson’s disease (WD) is a kind of inherited single-gene autosomal recessive disorder in which mutations in the ATP7B gene cause copper excretion disorders. Drug therapy is currently the main treatment method for WD. Liver transplantation should be considered for poor drug response or acute liver failure. However, it faces problems such as medication adherence, adverse reactions and shortage of liver source. Gene therapy in WD may permanently correct abnormal copper metabolism, which is why it is the focus of current research. This article summarizes the research progress of WD around gene therapy vectors and CRISPR/Cas9 gene editing system.