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103 results on '"Finn, Patrick"'

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1. Characterizing the mechanism of action for mRNA therapeutics for the treatment of propionic acidemia, methylmalonic acidemia, and phenylketonuria

2. mRNA therapy corrects defective glutathione metabolism and restores ureagenesis in preclinical argininosuccinic aciduria

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

4. The incidence of movement disorder increases with age and contrasts with subtle and limited neuroimaging abnormalities in argininosuccinic aciduria

5. Increasing Enzyme Mannose-6-Phosphate Levels but Not Miglustat Coadministration Enhances the Efficacy of Enzyme Replacement Therapy in Pompe Mice

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

8. Ex vivoprimary liver sections recapitulate disease phenotype and therapeutic rescue for liver monogenic diseases

9. Amnio acid substitution at position 298 of human glucose-6 phosphatase-α significantly impacts its stability in mammalian cells

11. mRNA therapy restores ureagenesis and corrects glutathione metabolism in argininosuccinic aciduria

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

16. mRNA therapy restores euglycemia and prevents liver tumors in murine model of glycogen storage disease

22. AssessBlocks

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

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

27. Mutation

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

42. Glycan Structure Determinants for Cation-Independent Mannose 6-Phosphate Receptor Binding and Cellular Uptake of a Recombinant Protein

46. Muscle Regeneration and Repair in the Pompe Mouse

47. Strategies for Neoglycan Conjugation to Human Acid α-Glucosidase

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

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