Abstract
Wilson disease is a rare disorder of copper metabolism caused by pathogenic variants in the ATP7B gene, which encodes a copper transporter, leading to toxic accumulation of copper in the liver, brain, and other organs. Wilson disease is progressive and can be fatal. Current standard of care includes copper chelators and zinc. However, these treatments have complex dose regimens and serious side effects. UX701 is an investigational AAV9 gene therapy designed to restore ATP7B function in the liver and is being developed for treatment of patients with Wilson disease.
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