Science advances · 2026
Restoring brain functions in the Mo-blo mouse model of Menkes disease with copper nanoclusters.
СтатьяНазвание и аннотация: Перевод в очереди
Обработка статьи · этапы, ошибки и управление
Загрузка состояния…
Ошибки и ограничения
История · было → стало
Состояние обновляется каждые 5 секунд, пока блок открыт. Процент показывается только там, где известен объём работы. Пауза останавливает продолжение этапа; текущий запрос к источнику может завершиться.
Malardé Audrey, Alberto Jean-Marc, Creusot Benjamin, Brice Laure, Paoli Justine, Julien Amélia, Umoret Rémy, Renard Pauline, Pourié Grégory, Sartelet Hervé, Arnoux Philippe, Barbault Florent, Brémond Eric, Jung-Rodriguez Emma, Courty Alexa, Walls Michael, Tizei Luiz, Madebène Bruno, Derat Etienne, Tremblay Benoit, Ouadi Ali, Brasse David, Coelho David, Wiedemann Arnaud, Meyre David, Clarot Igor, Feillet François, Boudier Ariane
Аннотация
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.