4.8 Article

Metabolic rescue ameliorates mitochondrial encephalo-cardiomyopathy in murine and human iPSC models of Leigh syndrome

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Summary: Leigh syndrome, a severe mitochondrial neurodegenerative disease, has no effective treatment yet. However, in a mouse model of Leigh syndrome, hypoxia treatment has shown promise in preventing neurodegeneration and significantly extending lifespan. Mild hypoxia alone may not be effective, but when combined with nicotinic acid supplementation, it can provide modest improvement in neurodegeneration and lifespan. Therapies targeting both brain hyperoxia and NAD(+) deficiency may hold promise for treating Leigh syndrome.

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