Article

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

2025-12-05

Abstract excerpt

The lysosomal storage disorder Fabry disease results from α-galactosidase A deficiency, leading to excessive glycosphingolipid substrate accumulation, primarily globotriaosylceramide (Gb3). While the underlying molecular mechanisms remain elusive, multi-systemic complications ultimately culminate in premature death, with heart failure being the leading cause of death. Current treatment options fail to treat Fabry...

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Literature Corpus work: a4be7ef9-bcab-5ab4-abd3-e9f98baf5f7a
DOI: 10.64898/2025.12.03.692072

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