GeneticsbioRxivPreprint
Experimental galactose-1 phosphate uridylyltransferase (GALT) mRNA therapy improves motor-related phenotypes in a mouse model of Classic Galactosemia
Olivia Bellagamba, Aaron J. Guo, Xinhua Yan…·Kent Lai
This study shows that experimental GALT mRNA therapy improves motor function in a mouse model of Classic Galactosemia.
DOI 10.1101/2025.04.21.649843