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Targeting liver nuclear receptors as a treatment for Wilsons disease

Wilson disease is a hepato-neurologic disorder caused by mutations in the gene ATP7B and accumulation of copper in tissues, predominantly in the liver. The disease is lethal, unless treated. Current life-long treatment involves copper chelation or copper replacement using Zn. Both procedures alleviate major symptoms but side effects are frequent and...

Published: 3/13/2025 | Inventor(s): Svetlana Lutsenko, James Hamilton

Keywords(s): Agonists/Promoters, Biomarker, Disease Indication, Genetic Disorders, Liver, Liver Transplant, Mechanism-of-action Biomarker, Non-novel, Off-the-shelf, Predicted Novelty, Single, Small Molecules, Targeted Therapy, Therapeutic Matter, Therapeutic Substance, Therapeutics, Therapy Type, Transplants, Wilson's Disease

Category(s): Clinical and Disease Specializations, Clinical and Disease Specializations > Genetic Diseases > Wilson's Disease, Clinical and Disease Specializations > Rare Diseases, Clinical and Disease Specializations > Transplantation > Liver Transplant, Technology Classifications > Diagnostics > Biomarkers, Technology Classifications > Therapeutic Modalities > Small Molecules, Technology Classifications > Therapeutic Modalities > Targets, Technology Classifications > Diagnostics, Clinical and Disease Specializations > Genetic Diseases, Technology Classifications > Therapeutic Modalities, Clinical and Disease Specializations > Transplantation