{
  "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.",
  "abstract_shown": true,
  "affiliations": [
    "Université de Lorraine, Inserm, NGERE, F-54000 Nancy, France.",
    "Université de Lorraine, CITHEFOR, F-54000 Nancy, France.",
    "Université de Lorraine, CNRS, LRGP, F-54000 Nancy, France.",
    "Department of Pathology, Nancy Regional University Hospital, F-54000 Nancy, France.",
    "Université Paris Cité, ITODYS, CNRS, F-75013 Paris, France.",
    "Sorbonne Université, MONARIS, CNRS-UMR 8233, F-75005 Paris, France.",
    "Laboratoire de Physique des Solides, Université Paris-Saclay, F-91405 Orsay, France.",
    "Sorbonne Université, IPCM, CNRS-UMR 8232, F-75005 Paris, France.",
    "Institut Pluridisciplinaire Hubert Curien, Université de Strasbourg, CNRS, UMR 7178, F-67037 Strasbourg, France.",
    "Pediatric Department Reference Center for Inborn Errors of Metabolism Children University Hospital Nancy, F-54000 Nancy, France.",
    "Nancy University Hospital, Department of Molecular Medicine, Division of Biochemistry, Molecular Biology, and Nutrition, F-54000 Nancy, France.",
    "Institut Universitaire de France (IUF), Paris, France."
  ],
  "article_type": null,
  "arxiv": null,
  "author_total": 28,
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    {
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        0
      ],
      "name": "Audrey Malardé",
      "orcid": "0009-0007-8452-2488"
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      "name": "Laure Brice",
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      "name": "Justine Paoli",
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      "name": "Amélia Julien",
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      "name": "Rémy Umoret",
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      "name": "Pauline Renard",
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      "name": "Grégory Pourié"
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      "name": "Hervé Sartelet",
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      "name": "Philippe Arnoux"
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      "name": "Florent Barbault",
      "orcid": "0000-0002-6082-3194"
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      "name": "Eric Brémond"
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      "name": "Emma Jung-Rodriguez",
      "orcid": "0009-0001-8175-5143"
    },
    {
      "affs": [
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      ],
      "name": "Alexa Courty",
      "orcid": "0000-0003-2742-018X"
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    {
      "affs": [
        6
      ],
      "name": "Michael Walls"
    },
    {
      "affs": [
        6
      ],
      "name": "Luiz Tizei",
      "orcid": "0000-0003-3998-9912"
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    {
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      ],
      "name": "Bruno Madebène",
      "orcid": "0000-0002-5058-5647"
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      "name": "Etienne Derat",
      "orcid": "0000-0002-8637-2707"
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    {
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      "name": "Benoit Tremblay",
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      "name": "Ali Ouadi",
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      "name": "David Brasse",
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      "name": "David Coelho",
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      "name": "Arnaud Wiedemann",
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      "name": "David Meyre",
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      "name": "Igor Clarot",
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      "name": "François Feillet",
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        2,
        11
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      "name": "Ariane Boudier",
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  "bibtex": "https://research.draconitic.com/paper/50y53epf9ny6e.bib",
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  "doi": "10.1126/sciadv.aeg6972",
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  "pdf_url": "https://hal.science/hal-05778254/document",
  "pmcid": null,
  "pmid": "42814820",
  "published": "2026-09-30",
  "publisher": "American Association for the Advancement of Science (AAAS)",
  "ris": "https://research.draconitic.com/paper/50y53epf9ny6e.ris",
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  "subjects": [
    {
      "field": "biology",
      "origin": "hal:0.sdv",
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  "title": "Restoring brain functions in the Mo-blo mouse model of Menkes disease with copper nanoclusters",
  "title_text": "Restoring brain functions in the Mo-blo mouse model of Menkes disease with copper nanoclusters",
  "type": "peer-reviewed",
  "updated_at": "2026-10-30T12:18:00Z",
  "url": "https://research.draconitic.com/paper/50y53epf9ny6e",
  "venue": "Science Advances",
  "versions": []
}