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Molecular Genetics and Metabolism|December 29, 2022
Synthetic mRNA rescues very long-chain acyl-CoA dehydrogenase deficiency in patient fibroblasts and a murine modelXue-Jun Zhao, Ai-Walid Mohsen, Stephanie Mihalik, et al.American Journal of Human Genetics|March 19, 2019
Systemic mRNA Therapy for the Treatment of Fabry Disease: Preclinical Studies in Wild-Type Mice, Fabry Mouse Model, and Wild-Type Non-human PrimatesXuling Zhu, Ling Yin, Matt Theisen, et al.Human Molecular Genetics|May 10, 2023
Messenger RNA rescues medium-chain acyl-CoA dehydrogenase deficiency in fibroblasts from patients and a murine modelXue-Jun Zhao, Ai-Walid Mohsen, Stephanie Mihalik, et al.Science Translational Medicine|January 10, 2024
mRNA therapy corrects defective glutathione metabolism and restores ureagenesis in preclinical argininosuccinic aciduriaSonam Gurung, Oskar Vilhelmsson Timmermand, Dany Perocheau, et al.Journal of Inherited Metabolic Disease|December 4, 2023
The incidence of movement disorder increases with age and contrasts with subtle and limited neuroimaging abnormalities in argininosuccinic aciduriaSonam Gurung, Saketh Karamched, Dany Perocheau, et al.Pageof 2