[Research progress in gene therapy for Wilson's disease].

ABSTRACT

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.