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, Joe Sarkis, Bijina Balakrishnan, Kent Lai
This study shows that experimental GALT mRNA therapy improves motor function in a mouse model of Classic Galactosemia.
bioRxiv · Apr 21, 2025 · PreprintGenetics