rAAV-ATP7B

**rAAV-ATP7B** is an investigational **gene therapy** for **Wilson disease** that uses a recombinant adeno-associated viral vector to deliver a functional **ATP7B** transgene to hepatocytes, with the goal of restoring hepatic copper transport and biliary copper excretion after a single administration. The therapeutic rationale is to replace deficient or dysfunctional ATP7B, the copper-transporting ATPase mutated in Wilson disease, thereby reducing toxic copper accumulation in the liver and other tissues. The term is most commonly used as a **nonproprietary preclinical descriptor** rather than a single uniquely branded clinical asset, and it has been used in the Wilson disease gene therapy literature and patent filings describing rAAV-mediated ATP7B replacement approaches. More specific clinical programs in this area have later appeared under distinct development code names rather than this exact generic construct name.

Other names

recombinant adeno-associated virus encoding ATP7BATP7B gene therapyATP-7B gene therapyATP 7B gene therapy