Finn, Patrick F.

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

Zhu, X., Yin, L., Theisen, M., Zhuo, J., Siddiqui, S., Levy, B., Presnyak, V., Frassetto, A., Milton, J., Salerno, T., et al. (2019) · The American Journal of Human Genetics

Lysosomal Storage Disorders ResearchGlycogen Storage Diseases and Myoclonus

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mRNA Therapy Improves Metabolic and Behavioral Abnormalities in a Murine Model of Citrin Deficiency

Cao, J., An, D., Galduroz, M., Zhuo, J., Liang, S., Eybye, M., Frassetto, A., Kuroda, E., Funahashi, A., Santana, J., et al. (2019) · Molecular Therapy

RNA Interference and Gene DeliveryMetabolism and Genetic Disorders

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