Article

Experimental galactose-1 phosphate uridylyltransferase (GALT) mRNA therapy improves motor-related phenotypes in a mouse model of Classic Galactosemia

2025-04-24

Abstract excerpt

Despite life-saving newborn screening programs and a life-long galactose-restricted diet, many patients with Classic Galactosemia continue to develop long-term debilitating neurological deficits, speech dyspraxia, and primary ovarian insufficiency (POI). Earlier, we showed that administration of an experimental human GALT mRNA predominantly expressed in the liver of the GalT gene-trapped mouse model augmented th...

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Literature Corpus work: a0062331-3408-55b3-ae11-c40f662372ed
DOI: 10.1101/2025.04.21.649843

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Experimental galactose-1 phosphate uridylyltransferase (GALT) mRNA therapy improves motor-related phenotypes in a mouse model of Classic GalactosemiaDOI 10.1101/2025.04.21.649843Read

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