Gene therapy of Wilson disease: a “golden” opportunity using rAAV on the 50th anniversary of the discovery of the virus

Wilson disease (WD) was a once progressive and uniformly fatal inherited disorder of copper metabolism. Medical therapy to arrest progression or prevent complications of WD was developed in the 1950’s with the introduction of parenterally administered BAL[1], and over the next two decades by oral therapy with d-penicillamine trientine, and zinc [2]. Effective therapy for WD requires life-long administration of daily medication. At least 30-50% of patients on medication for their WD have periods of non-adherence, some suffering liver failure, others developing potentially irreversible neurologic or psychiatric symptoms.

Source: Journal of Hepatology - November 27, 2015 Category: Gastroenterology Authors: Jayanta Roy-Chowdhury, Michael L. Schilsky Source Type: research