Nucleoside-Modified mRNA Encoding Alpha-Galactosidase A Reverses Fabry Disease Phenotypes in Human IPSC-Derived Cardiomyocytes
Malte Juchem, Sedef Ersoy, Lea Oehlsen, Jia Li Ye, Natalie Weber, Ke Xiao, Christopher Jahn, Reto Eggenschwiler, Tobias Cantz, Julia Beimdiek, Falk F. R. Buettner, Theresia Kraft, Jan Hegermann, Christian Bär, Jeannine Hoepfner, Thomas Thum
This study shows that modGLA mRNA therapy reverses Fabry disease phenotypes in human iPSC-derived cardiomyocytes by restoring enzyme activity and normalizing molecular alterations.
bioRxiv · Dec 5, 2025 · PreprintMolecular Biology