268 results on '"Scheule, Ronald"'
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2. The safety profile of a cationic lipid-mediated cystic fibrosis gene transfer agent following repeated monthly aerosol administration to sheep
3. Efficacy of Enzyme and Substrate Reduction Therapy with a Novel Antagonist of Glucosylceramide Synthase for Fabry Disease
4. Secreted Gaussia luciferase as a sensitive reporter gene for in vivo and ex vivo studies of airway gene transfer
5. Distribution of acid sphingomyelinase in rodent and non-human primate brain after intracerebroventricular infusion
6. The use of carboxymethylcellulose gel to increase non-viral gene transfer in mouse airways
7. The role of doxorubicin in non-viral gene transfer in the lung
8. Rapid identification of novel functional promoters for gene therapy
9. In vivo imaging of gene transfer to the respiratory tract
10. Improved management of lysosomal glucosylceramide levels in a mouse model of type 1 Gaucher disease using enzyme and substrate reduction therapy
11. Endostatin gene transfection using a cationic lipid: advantages of transfection before tumor cell inoculation and repeated transfection
12. Assessment of the nuclear pore dilating agent trans-cyclohexane-1,2-diol in differentiated airway epithelium
13. The ΔF508 CFTR defect: molecular mechanism of suppressor mutation V510D and the contribution of transmembrane helix unraveling
14. Limitations of the Murine Nose in the Development of Nonviral Airway Gene Transfer
15. Surfactant Protein-D Inhibits Lung Inflammation Caused by Ventilation in Premature Newborn Lambs
16. Toxicity of Cationic Lipid‐DNA Complexes
17. Surfactant Protein-D and Surfactant Inhibit Endotoxin-Induced Pulmonary Inflammation*
18. Intratracheal Recombinant Surfactant Protein D Prevents Endotoxin Shock in the Newborn Preterm Lamb
19. Cationic Lipid-Mediated Gene Delivery to the Airways
20. [52] Cationic lipid formulations for intracellular gene delivery of cystic fibrosis transmembrane conductance regulator to airway epithelia
21. Transforming growth factor–β1 gene transfer ameliorates acute lung allograft rejection
22. EX VIVO TRANSFECTION OF TRANSFORMING GROWTH FACTOR-β1 GENE TO PULMONARY ARTERY SEGMENTS IN LUNG GRAFTS
23. LIPOSOME-MEDIATED GENE TRANSFER IN RAT LUNG TRANSPLANTATION: A COMPARISON BETWEEN THE IN VIVO AND EX VIVO APPROACHES
24. EX VIVO LIPOSOME-MEDIATED GENE TRANSFER TO LUNG ISOGRAFTS
25. LIPOSOME-MEDIATED GENE TRANSFER TO LUNG ISOGRAFTS
26. SUCCESSFUL IN VIVO AND EX VIVO TRANSFECTION OF PULMONARY ARTERY SEGMENTS IN LUNG ISOGRAFTS
27. Role of viral hemagglutinin glycosylation in anti-influenza activities of recombinant surfactant protein D
28. Inefficient cationic lipid-mediated siRNA and antisense oligonucleotide transfer to airway epithelial cells in vivo
29. Modification of Asbestos Bioactivity for the Alveolar Macrophage by Selective Protein Adsorption
30. The role of CpG motifs in immunostimulation and gene therapy
31. Transfection of pulmonary artery segments in lung isografts during storage
32. Ex vivo transfection of pulmonary artery segments in lung isografts
33. A randomised, double-blind, placebo-controlled trial of repeated nebulisation of non-viral cystic fibrosis transmembrane conductance regulator (CFTR) gene therapy in patients with cystic fibrosis
34. Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial
35. Antisense oligonucleotide-mediated suppression of muscle glycogen synthase 1 synthesis as an approach for substrate reduction therapy of Pompe disease
36. Antisense Oligonucleotide-mediated Suppression of Muscle Glycogen Synthase 1 Synthesis as an Approach for Substrate Reduction Therapy of Pompe Disease
37. Dysregulation of Multiple Facets of Glycogen Metabolism in a Murine Model of Pompe Disease
38. A novel, selective and orally-available glucosylceramide synthase inhibitor for substrate reduction therapy of Fabry disease
39. Systemic Delivery of a Glucosylceramide Synthase Inhibitor Reduces CNS Substrates and Increases Lifespan in a Mouse Model of Type 2 Gaucher Disease
40. Iminosugar-based inhibitors of glucosylceramide synthase prolong survival but paradoxically increase brain glucosylceramide levels in Niemann–Pick C mice
41. Glucosylceramide Synthase Inhibition Reduces Gb3 and Lyso-Gb3 in a Mouse Model Of Fabry Disease
42. Pompe Syndrome: Dysregulation of Multiple Facets of Glycogen Metabolism in a Murine Model of Pompe Disease
43. Systemic Administration of AAV8-α-galactosidase A Induces Humoral Tolerance in Nonhuman Primates Despite Low Hepatic Expression
44. Iminosugar-Based Inhibitors of Glucosylceramide Synthase Increase Brain Glycosphingolipids and Survival in a Mouse Model of Sandhoff Disease
45. Inhibiting glycogen biosynthesis by mTORC1 suppression as an adjunct therapy for Pompe disease
46. IgG Specifically Enhances Chrysotile Asbestos-stimulated Superoxide Anion Production by the Alveolar Macrophage
47. Induction of Immune Tolerance to a Therapeutic Protein by Intrathymic Gene Delivery
48. Substrate Reduction Augments the Efficacy of Enzyme Therapy in a Mouse Model of Fabry Disease
49. Preexisting Immunity and Low Expression in Primates Highlight Translational Challenges for Liver-directed AAV8-mediated Gene Therapy
50. Evaluation of Systemic Follistatin as an Adjuvant to Stimulate Muscle Repair and Improve Motor Function in Pompe Mice
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