UX701 is an experimental gene therapy for

Wilson disease Wilson's disease (also called hepatolenticular degeneration) is a genetic disorder characterized by the excess build-up of copper in the body. Symptoms are typically related to the brain and liver. Liver-related symptoms include vomiting, weak ...

, delivered via

adeno-associated virus Adeno-associated viruses (AAV) are small viruses that infect humans and some other primate species. They belong to the genus '' Dependoparvovirus'', which in turn belongs to the family ''Parvoviridae''. They are small (approximately 26 nm in ...

, that restores a functional version of the

ATP7B Wilson disease protein (WND), also known as ATP7B protein, is a copper-transporting P-type ATPase which is encoded by the ''ATP7B'' gene. The ATP7B protein is located in the trans-Golgi network of the liver and brain and balances the copper l ...

gene.

References

{{reflist Experimental gene therapies