Gene therapy of Wilson disease: A “golden” opportunity using rAAV on the 50th anniversary of the discovery of the virus
J Roy-Chowdhury, ML Schilsky - Journal of Hepatology, 2016 - journal-of-hepatology.eu
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 1950s 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 WD have periods of nonadherence, some suffering liver …
copper metabolism. Medical therapy to arrest progression or prevent complications of WD
was developed in the 1950s 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 WD have periods of nonadherence, some suffering liver …