94 results on '"Scheule, Ronald"'
Search Results
2. The ΔF508 CFTR defect: molecular mechanism of suppressor mutation V510D and the contribution of transmembrane helix unraveling
3. Toxicity of Cationic Lipid‐DNA Complexes
4. Cationic Lipid-Mediated Gene Delivery to the Airways
5. [52] Cationic lipid formulations for intracellular gene delivery of cystic fibrosis transmembrane conductance regulator to airway epithelia
6. Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial
7. Antisense oligonucleotide-mediated suppression of muscle glycogen synthase 1 synthesis as an approach for substrate reduction therapy of Pompe disease
8. Efficacy of Enzyme and Substrate Reduction Therapy with a Novel Antagonist of Glucosylceramide Synthase for Fabry Disease
9. Antisense Oligonucleotide-mediated Suppression of Muscle Glycogen Synthase 1 Synthesis as an Approach for Substrate Reduction Therapy of Pompe Disease
10. The safety profile of a cationic lipid-mediated cystic fibrosis gene transfer agent following repeated monthly aerosol administration to sheep
11. Dysregulation of Multiple Facets of Glycogen Metabolism in a Murine Model of Pompe Disease
12. A novel, selective and orally-available glucosylceramide synthase inhibitor for substrate reduction therapy of Fabry disease
13. Systemic Delivery of a Glucosylceramide Synthase Inhibitor Reduces CNS Substrates and Increases Lifespan in a Mouse Model of Type 2 Gaucher Disease
14. Rapid identification of novel functional promoters for gene therapy
15. Assessment of the nuclear pore dilating agent trans‐cyclohexane‐1,2‐diol in differentiated airway epithelium
16. Iminosugar-based inhibitors of glucosylceramide synthase prolong survival but paradoxically increase brain glucosylceramide levels in Niemann–Pick C mice
17. Glucosylceramide Synthase Inhibition Reduces Gb3 and Lyso-Gb3 in a Mouse Model Of Fabry Disease
18. Pompe Syndrome: Dysregulation of Multiple Facets of Glycogen Metabolism in a Murine Model of Pompe Disease
19. Systemic Administration of AAV8-α-galactosidase A Induces Humoral Tolerance in Nonhuman Primates Despite Low Hepatic Expression
20. Distribution of acid sphingomyelinase in rodent and non-human primate brain after intracerebroventricular infusion
21. Iminosugar-Based Inhibitors of Glucosylceramide Synthase Increase Brain Glycosphingolipids and Survival in a Mouse Model of Sandhoff Disease
22. Secreted Gaussia luciferase as a sensitive reporter gene for in vivo and ex vivo studies of airway gene transfer
23. Inhibiting glycogen biosynthesis by mTORC1 suppression as an adjunct therapy for Pompe disease
24. Induction of Immune Tolerance to a Therapeutic Protein by Intrathymic Gene Delivery
25. Substrate Reduction Augments the Efficacy of Enzyme Therapy in a Mouse Model of Fabry Disease
26. Preexisting Immunity and Low Expression in Primates Highlight Translational Challenges for Liver-directed AAV8-mediated Gene Therapy
27. Evaluation of Systemic Follistatin as an Adjuvant to Stimulate Muscle Repair and Improve Motor Function in Pompe Mice
28. Inhibition of glycogen biosynthesis via mTORC1 suppression as an adjunct therapy for Pompe disease
29. Limitations of the Murine Nose in the Development of Nonviral Airway Gene Transfer
30. Surfactant Protein-D Inhibits Lung Inflammation Caused by Ventilation in Premature Newborn Lambs
31. Improved management of lysosomal glucosylceramide levels in a mouse model of type 1 Gaucher disease using enzyme and substrate reduction therapy
32. The use of carboxymethylcellulose gel to increase non-viral gene transfer in mouse airways
33. 90. Efficacy of Genz-529468-mediated inhibition of glucosylceramide synthase in a mouse model of Sandhoff disease
34. 91. Management of lysosomal glycosphingolipid levels in animal models of Gaucher and Fabry disease with enzyme and substrate reduction therapies
35. Low-frequency ultrasound increases non-viral gene transfer to the mouse lung
36. Detection of CFTR transgene mRNA expression in respiratory epithelium isolated from the murine nasal cavity
37. Surfactant protein-D regulates the postnatal maturation of pulmonary surfactant lipid pool sizes
38. The role of doxorubicin in non-viral gene transfer in the lung
39. Role of viral hemagglutinin glycosylation in anti-influenza activities of recombinant surfactant protein D
40. Systemic Insulin-like Growth Factor-1 Reverses Hypoalgesia and Improves Mobility in a Mouse Model of Diabetic Peripheral Neuropathy
41. Ability of Adeno-Associated Virus Serotype 8-Mediated Hepatic Expression of Acid α-Glucosidase to Correct the Biochemical and Motor Function Deficits of Presymptomatic and Symptomatic Pompe Mice
42. In vivo imaging of gene transfer to the respiratory tract
43. Surfactant Protein-D and Surfactant Inhibit Endotoxin-Induced Pulmonary Inflammation
44. Timing of Therapeutic Intervention Determines Functional and Survival Outcomes in a Mouse Model of Late Infantile Batten Disease
45. Intratracheal Recombinant Surfactant Protein D Prevents Endotoxin Shock in the Newborn Preterm Lamb
46. Inefficient cationic lipid-mediated siRNA and antisense oligonucleotide transfer to airway epithelial cells in vivo
47. 475. SeV-Mediated Gene Transfer after Three Re-Administrations in Mice and Sheep
48. 217. Comparison of Hepatic Vein and Peripheral Vein Delivery of an AAV2/8 Vector Encoding Human a-Galactosidase A to Rhesus Macaques
49. 893. Second Generation Gene Therapy Vector for Classical Late Infantile Neuronal Ceroid Lipofuscinosis
50. 695. Low-Frequency Ultrasound Increases Non- Viral Lung Gene Transfer
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