Modified messenger RNA shows potential as treatment for Fabry disease in stem cells

Embargoed until: Publicly released: 2023-08-22 01:00

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Researchers at Murdoch Children's Research Institute have led a collaboration demonstrating that modified messenger RNA (modRNA) shows great potential as a treatment for Fabry disease. In this study, the team both validated the use of iPSC-derived heart muscle cells from affected individuals as a model to study disease processes in Fabry disease and showed that the modRNA treatment was found to effectively decrease the harmful buildup of glycosphingolipid Globotriaosylceramide (GB3) in the heart. These findings advance a potential therapy for Fabry disease.

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Research Cell Press, Web page

Journal/
conference: The American Journal of Human Genetics

Research:Paper

Organisation/s: Murdoch Children's Research Institute (MCRI), The University of Melbourne, QIMR Berghofer Medical Research Institute, Queensland University of Technology (QUT), The University of Adelaide, Monash University

Funder: We acknowledge grant and fellowship support from the National Health and Medical Research Council of Australia (E.R.P., D.A.E.), Australian Research Council (E.R.P.), Heart Foundation of Australia (E.R.P., D.A.E.), The Medical Research Future Fund (E.R.P., D.A.E.), The Stafford Fox Medical Research Foundation (E.R.P.), and the Royal Children’s Hospital Foundation (E.R.P., D.A.E.). MCRI is supported by the Victorian Government’s Opera- Q12 tional Infrastructure Support Program. E.R.P. and D.A.E. are Principal Investigators of The Novo Nordisk Foundation Center for Stem Cell Medicine (Novo Nordisk Foundation grant number NNF21CC0073729).

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