Detalle Publicación


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

Título de la revista: JOURNAL OF HEPATOLOGY
ISSN: 1600-0641
Volumen: 64
Número: 2
Páginas: 419-26
Fecha de publicación: 2016
Our data demonstrate that AAV8-AAT-ATP7B-mediated gene therapy provides long-term correction of copper metabolism in a clinically relevant animal model of WD providing support for future translational studies.