A liver-targeted AAV8-mediated ATP7B (AAV8-ΔC4ATP7B) gene therapy rescued impaired Cu metabolism and reversed liver histopathology without adverse effects in Atp7b R780L knockin (KI) mouse model, supporting the potential of AAV8-ΔC4ATP7B as a safe and effective treatment for Wilson’s disease. This evidence concerns the gene ATP7B and Wilson disease.