Restoring brain functions in the Mo-blo mouse model of Menkes disease with copper nanoclusters.

Authors

Audrey Malardé, Jean-Marc Alberto, Benjamin Creusot, Laure Brice, Justine Paoli, Amélia Julien, Rémy Umoret, Pauline Renard, Grégory Pourié, Hervé Sartelet, Philippe Arnoux, Florent Barbault, Eric Brémond, Emma Jung-Rodriguez, Alexa Courty, Michael Walls, Luiz Tizei, Bruno Madebène, Etienne Derat, Benoit Tremblay, Ali Ouadi, David Brasse, David Coelho, Arnaud Wiedemann, David Meyre, Igor Clarot, François Feillet, Ariane Boudier

Published in

Science advances. Volume 12. Issue 40. Pages eaeg6972. Oct 02, 2026. Epub Sep 30, 2026.

Abstract

Menkes disease, caused by ATP7A mutations, leads to severe copper deficiency and fatal neurodegeneration. Current copper-histidine therapy fails to restore brain function. Copper nanoclusters (CuNCs) were designed for their ultrasmall size to cross physiological barriers and deliver copper directly to the whole organism, including the brain. In Mo-blo mice (ATP7A deficient), daily subcutaneous CuNCs markedly improved survival, normalized cuproprotein activities, and restored cognitive and motor functions. Unlike other treatments, CuNCs effectively penetrate the blood-brain barrier, deliver copper to cuproproteins, offering unprecedented neurological rescue. This breakthrough could transform Menkes disease treatment, providing a first-ever therapy for brain copper deficiency. Further clinical development may unlock lifesaving solutions for this rare, fatal disorder.

PMID:
42814820
Bibliographic data and abstract were imported from PubMed on 01 Oct 2026.

Read full publication at:
Please sign in to see all details.

Stats

1-terrible, 9-excellent. How would you rate this publication? Sign in in to submit your rating.

Post a comment

You need to be signed in to post comments. You can sign in here.

Comments

There are no comments yet.