Long-term metabolic correction of Wilson's disease in a murine model by gene therapy

O Murillo, DM Luqui, C Gazquez… - Journal of …, 2016 - Elsevier
Background & Aims Wilson's disease (WD) is an autosomal recessively inherited copper
storage disorder due to mutations in the ATP7B gene that causes hepatic and neurologic
symptoms. Current treatments are based on lifelong copper chelating drugs and zinc salts,
which may cause side effects and do not restore normal copper metabolism. In this work we
assessed the efficacy of gene therapy to treat this condition. Methods We transduced the
liver of the Atp7b−/− WD mouse model with an adeno-associated vector serotype 8 (AAV8) …