Molecular BiologybioRxivPreprint
Nucleoside-Modified mRNA Encoding Alpha-Galactosidase A Reverses Fabry Disease Phenotypes in Human IPSC-Derived Cardiomyocytes
Malte Juchem, Sedef Ersoy, Lea Oehlsen…·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.
DOI 10.64898/2025.12.03.692072