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23 results on '"Finn, Patrick"'

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1. Whole-body galactose oxidation as a robust functional assay to assess the efficacy of gene-based therapies in a mouse model of Galactosemia

2. GLA-modified RNA treatment lowers GB3 levels in iPSC-derived cardiomyocytes from Fabry-affected individuals

4. Synthetic human ABCB4 mRNA therapy rescues severe liver disease phenotype in a BALB/c.Abcb4 mouse model of PFIC3

6. "Spontaneous" late recovery from stuttering: Dimensions of reported techniques and causal attributions

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

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

9. Muscle Regeneration and Repair in the Pompe Mouse

10. Inhibiting glycogen biosynthesis by mTORC1 suppression as an adjunct therapy for Pompe disease

12. Inhibition of glycogen biosynthesis via mTORC1 suppression as an adjunct therapy for Pompe disease

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