Abstract
Selection of the month Gene therapy for Wilson disease Current treatments for Wilson Disease (WD) are based on lifelong copper chelating drugs, which may cause side effects and do not restore normal copper metabolism. In this work, Murillo et al. assessed the efficacy of gene therapy by transducing the liver of the Atp7b −/− WD mouse model with an adenovirus encoding the human ATP7B cDNA. They observed a dose-dependent therapeutic effect of the gene therapy approach manifested by the reduction of serum transaminases and urinary copper excretion , normalization of serum holoceruloplasmin, and restoration of physiological biliary copper excretion in response to copper overload. The liver of treated animals showed normalization of copper content and absence of histological alterations. These promising preclinical studies highlight the potential of gene therapy to treat patients with WD.
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