Liver Expression of a MiniATP7B Gene Results in Long‐Term Restoration of Copper Homeostasis in a Wilson Disease Model in Mice
Oihana Murillo, Daniel Moreno, Cristina Gazquez, Miren Barberia, Itziar Cenzano, Iñigo Navarro, Iker Uriarte, Victor Sebastian, Manuel Arruebo, Veronica Ferrer, Bernard Bénichou, Jean Philippe Combal, Jesus Prieto, Ruben Hernandez‐Alcoceba, Gloria Gonzalez Aseguinolaza – 1 February 2019 – Gene therapy with an adeno‐associated vector (AAV) serotype 8 encoding the human ATPase copper‐transporting beta polypeptide (ATP7B) complementary DNA (cDNA; AAV8‐ATP7B) is able to provide long‐term copper metabolism correction in 6‐week‐old male Wilson disease (WD) mice.