Wilson's Disease: An Update

Shyamal K Das; Kunal Ray

Disclosures

Nat Clin Pract Neurol. 2006;2(9):482-493. 

In This Article

Conclusion

Despite significant advances in our understanding of the disease process in WD, further insights into the intracellular processes involved in copper homeostasis would enhance our knowledge and allow for improved treatment. Reported variations in the disease phenotype of related patients with the same set of mutations in the ATP7B gene suggest that the phenotype can be modulated by modifier genes such as ATOX1 and COMMD1. New methods that provide faster and cheaper means of genotyping patients will speed up the process of confirmation of diagnosis in targeted population groups, and DNA microarray analysis may help in the direct detection of the mutations. In terms of therapy, long-term clinical trials of recently advocated agents such as tetrathiomolybdate are needed to judge these treatments' efficacy, and a more comprehensive solution could involve gene therapy. To date, successful but only transient therapeutic effects of gene therapy have been reported in a rat model of WD using an adenoviral gene transfer vector.[62]


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