Nucleoside-Modified mRNA Encoding Alpha-Galactosidase A Ameliorates Fabry Disease Phenotypes in Human IPSC-Derived Cardiomyocytes.

Authors: Juchem M, Oehlsen L, Ersoy S, Ye JL, Weber N, Mohr E, Agyapong W, Liu J, Fuchs M, Xiao K, Jahn C, Eggenschwiler R, Cantz T, Beimdiek J, Buettner FFR, Kraft T, Hegermann J, Knorr IJ, Ernst L, Feldbrügge L, Puffert R, Schmidt K, Bär C, Hoepfner J, Thum T

Journal: Advanced science (Weinheim, Baden-Wurttemberg, Germany)