Skip to content
DraconiticResearch
Search Sources API
201,818 studies31 sourcesUpdated 10 min ago
Peer-reviewedCC BY 4.0Science Advances

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

  • Audrey Malardé1
  • Jean-Marc Alberto1
  • Benjamin Creusot2,3
  • Laure Brice2,3
  • Justine Paoli1
  • Amélia Julien1
  • Rémy Umoret1
  • Pauline Renard1
  • Grégory Pourié1
  • Hervé Sartelet1,4
  • Philippe Arnoux3
  • Florent Barbault5
16 more authors
  • Eric Brémond5
  • Emma Jung-Rodriguez5
  • Alexa Courty6
  • Michael Walls7
  • Luiz Tizei7
  • Bruno Madebène6
  • Etienne Derat8
  • Benoit Tremblay6
  • Ali Ouadi9
  • David Brasse9
  • David Coelho1,10
  • Arnaud Wiedemann1,10
  • David Meyre1,11
  • Igor Clarot2,3
  • François Feillet1,10
  • Ariane Boudier2,3,12
12 affiliations
  1. 1Université de Lorraine, Inserm, NGERE, F-54000 Nancy, France.
  2. 2Université de Lorraine, CITHEFOR, F-54000 Nancy, France.
  3. 3Université de Lorraine, CNRS, LRGP, F-54000 Nancy, France.
  4. 4Department of Pathology, Nancy Regional University Hospital, F-54000 Nancy, France.
  5. 5Université Paris Cité, ITODYS, CNRS, F-75013 Paris, France.
  6. 6Sorbonne Université, MONARIS, CNRS-UMR 8233, F-75005 Paris, France.
  7. 7Laboratoire de Physique des Solides, Université Paris-Saclay, F-91405 Orsay, France.
  8. 8Sorbonne Université, IPCM, CNRS-UMR 8232, F-75005 Paris, France.
  9. 9Institut Pluridisciplinaire Hubert Curien, Université de Strasbourg, CNRS, UMR 7178, F-67037 Strasbourg, France.
  10. 10Pediatric Department Reference Center for Inborn Errors of Metabolism Children University Hospital Nancy, F-54000 Nancy, France.
  11. 11Nancy University Hospital, Department of Molecular Medicine, Division of Biochemistry, Molecular Biology, and Nutrition, F-54000 Nancy, France.
  12. 12Institut Universitaire de France (IUF), Paris, France.
Open at sourcePDFDOICite

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.

Related

  1. Restoring Aquaporin-4 alleviates molecular and motor phenotypes in zQ175 mouse model of Huntington disease
    H. Liu, Z. Ding, Y. Wang +8
    bioRxivPreprintAll rights reservedFull textNeurosciencebioRxiv
  2. Restoring multiple TDP-43 cryptic targets, but not solely Unc13a, rescues motor neuron disease
    A. Peethambaran Mallika, M. S. Baghel, O. Sitzman +7
    bioRxivPreprintCC BY-NC-NDFull textNeurosciencebioRxiv
  3. CTR1-mediated copper uptake orchestrates metabolic-epigenetic regulation of pathogenic TH17 cells in autoimmune disease
    L. Noyer, L. Wang, M. Jishage +7
    bioRxivPreprintAll rights reservedFull textImmunologybioRxiv
  4. Exploring the biochemical and biological functions of copper radical oxidases in the vascular wilt phytopathogen Verticillium dahliae
    Jessica Fong, Ying-Yu Chen, Rebekka Harting +9
    Applied and Environmental MicrobiologyPeer-reviewedCC BY 4.0Full text1 citedCell biologyHAL
  5. How Periodontal Disease Compromises Oral Functions Related to Ingestion: Evidence From Systematic Review and Meta‐Analysis
    Thomas Douillard, Martine Hennequin, Emmanuel Nicolas +1
    Journal of Oral RehabilitationPeer-reviewedLicence not statedFull textGeneral biologyHAL

Cite

BibTeXRIS
BibTeX
@article{malarde2026restoring,
  title = {{Restoring brain functions in the Mo-blo mouse model of Menkes disease with copper nanoclusters}},
  author = {Audrey Malardé and Jean-Marc Alberto and Benjamin Creusot and Laure Brice and Justine Paoli and Amélia Julien and Rémy Umoret and Pauline Renard and Grégory Pourié and Hervé Sartelet and Philippe Arnoux and Florent Barbault and Eric Brémond and Emma Jung-Rodriguez and Alexa Courty and Michael Walls and Luiz Tizei and Bruno Madebène and Etienne Derat and Benoit Tremblay and Ali Ouadi and David Brasse and David Coelho and Arnaud Wiedemann and David Meyre and Igor Clarot and François Feillet and Ariane Boudier},
  journal = {Science Advances},
  year = {2026},
  month = sep,
  doi = {10.1126/sciadv.aeg6972},
  url = {https://www.science.org/doi/10.1126/sciadv.aeg6972},
  note = {CC BY 4.0},
}
RIS
TY  - JOUR
TI  - Restoring brain functions in the Mo-blo mouse model of Menkes disease with copper nanoclusters
AU  - Audrey Malardé
AU  - Jean-Marc Alberto
AU  - Benjamin Creusot
AU  - Laure Brice
AU  - Justine Paoli
AU  - Amélia Julien
AU  - Rémy Umoret
AU  - Pauline Renard
AU  - Grégory Pourié
AU  - Hervé Sartelet
AU  - Philippe Arnoux
AU  - Florent Barbault
AU  - Eric Brémond
AU  - Emma Jung-Rodriguez
AU  - Alexa Courty
AU  - Michael Walls
AU  - Luiz Tizei
AU  - Bruno Madebène
AU  - Etienne Derat
AU  - Benoit Tremblay
AU  - Ali Ouadi
AU  - David Brasse
AU  - David Coelho
AU  - Arnaud Wiedemann
AU  - David Meyre
AU  - Igor Clarot
AU  - François Feillet
AU  - Ariane Boudier
PY  - 2026
DA  - 2026/09/30
JO  - Science Advances
DO  - 10.1126/sciadv.aeg6972
UR  - https://www.science.org/doi/10.1126/sciadv.aeg6972
AB  - 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.
SN  - 2375-2548
ER  -