High value of 64Cu as a tool to evaluate the restoration of physiological copper excretion after gene therapy in Wilson’s disease

High value of 64Cu as a tool to evaluate the restoration of physiological copper excretion after gene therapy in Wilson’s disease

Wilson’s disease (WD) is an inherited disorder of copper metabolism associated with mutations in ATP7B gene. We have shown that the administration of an adeno-associated vector (AAV) encoding a mini version of human ATP7B (VTX-801) provides long-term correction of copper metabolism in a murine WD mo...

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Bibliographic Details
Main Authors: Oihana Murillo, Maria Collantes, Cristina Gazquez, Daniel Moreno, Ruben Hernandez-Alcoceba, Miren Barberia, Margarita Ecay, Blanche Tamarit, Anne Douar, Veronica Ferrer, Jean Philippe Combal, Ivan Peñuelas, Bernard Bénichou, Gloria Gonzalez-Aseguinolaza
Format: Article
Language:English
Published: Elsevier 2022-09-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
Wilson’s disease
radiocopper
AAV
ATP7B
copper metabolism
gene transfer
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050122000791

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http://www.sciencedirect.com/science/article/pii/S2329050122000791

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