LY-M003

Wilson's disease (WD), also known as Wilson's disease, is a rare autosomal recessive metabolic disorder caused by a mutation of the copper transport ATPase β (ATP7B) gene located on the long arm of chromosome 13 (13q14.3). This leads to accumulation of copper ions in multiple organs such as liver, brain and kidney, resulting in organ involvement. In this study, rAAV8 as the carrier of gene therapy products. After a single intravenous infusion, ATP7B protein can be specifically transduced to the target organ liver and expressed in hepatocytes for a long time.