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

Copper nanoclusters enable complete neurological rescue in Mo-blo mice, a model of Menkes disease, by crossing the blood-brain barrier and restoring brain copper levels.

• Why it matters: Menkes disease causes severe neurodegeneration due to copper deficiency, and current treatments fail to effectively deliver copper to the brain, leaving a critical therapeutic gap.
• What they did: Researchers designed ultrasmall copper nanoclusters and administered them subcutaneously to Mo-blo mice, a model lacking ATP7A, to evaluate their ability to restore copper-dependent functions.
• The result: CuNCs significantly improved survival, normalized enzyme activities, and fully restored cognitive and motor functions, demonstrating a promising approach for treating brain copper deficiency in Menkes disease.