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.
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a callDisclaimer: All third-party content on this website/platform is and will remain the property of their respective owners and is provided on "as is" basis without any warranties, express or implied. Use of third-party content does not indicate any affiliation, sponsorship with or endorsement by them. Any references to third-party content is to identify the corresponding services and shall be considered fair use under The CopyrightLaw.
