Pilot Clinical Study Showing Abnormal Copper Metabolism in Healthy Wilson Disease Heterozygote Subjects
Benichou B, Combal J, Dogterom P, Sandahl T, D'Antiga L, Schilsky M. Pilot Clinical Study Showing Abnormal Copper Metabolism in Healthy Wilson Disease Heterozygote Subjects. Clinical And Translational Science 2025, 18: e70294. PMCID: PMC12271968, DOI: 10.1111/cts.70294.Peer-Reviewed Original ResearchHealthy volunteersGene therapyOpen-label pilot clinical studyPotential dimerizationGene repair therapyCu-transporting P-type ATPaseLife-threatening disorderDominant negative effectP-type ATPasesPilot clinical studyAutosomal recessive mannerAbnormal copper metabolismBiliary copper excretionMutant proteinsIntravenous doseCu-dependent enzymesATP7B mutationsClinical studiesATP7B functionCopper excretionRecessive mannerHeterozygote subjectsTherapyBiosynthetic incorporationTransport proteins
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