Gene therapy for Wilson's Disease: AAV-ATP7B

  • Wilson's Disease is a rare autosomal recessive inborn error of copper metabolism caused by mutations in ATP7B, which encodes a copper-transporting adenosine triphosphatase.

  • Current treatment options are only moderately effective and the only curative treatment is liver transplantation thus there is a need to develop long‐term effective treatments.

  • Adenoassociated viral (AAV) vectors are very promising tools for therapeutic gene delivery, since they are safe and they induce an efficient and long-term transduction.

  • A new AAV vector, AAV-ATP7B, expressing the therapeutic gene ATP7B has been developed and tested in a Wilson's disease animal models.

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