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2019•The American Journal of Human GeneticsOpen access

Systemic mRNA Therapy for the Treatment of Fabry Disease: Preclinical Studies in Wild-Type Mice, Fabry Mouse Model, and Wild-Type Non-human Primates

Xuling Zhu, Ling Yin, Matt Theisen, Jenny Zhuo, Summar Siddiqui, Becca R. Levy, Vladimir Presnyak, Andrea Frassetto, Jaclyn Milton, Timothy Salerno, Kerry E. Benenato, Joe Milano, Andy Lynn, Staci Sabnis, Kristine Burke, Gilles Besin, Christine Lukacs, Lin T. Guey, Patrick F. Finn, Paolo G.V. Martini

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Key concepts: Fabry disease, Globotriaosylceramide, Messenger RNA, Enzyme replacement therapy, Lysosomal storage disease, Wild type, Medicine, Disease

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Systemic mRNA Therapy for the Treatment of Fabry Disease: Preclinical Studies in Wild-Type Mice, Fabry Mouse Model, and Wild-Type Non-human Primates — Research Paper | ScholarLens