182 results on '"Scheule, Ronald"'
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2. Efficacy of Enzyme and Substrate Reduction Therapy with a Novel Antagonist of Glucosylceramide Synthase for Fabry Disease
3. Secreted Gaussia luciferase as a sensitive reporter gene for in vivo and ex vivo studies of airway gene transfer
4. The use of carboxymethylcellulose gel to increase non-viral gene transfer in mouse airways
5. The role of doxorubicin in non-viral gene transfer in the lung
6. Rapid identification of novel functional promoters for gene therapy
7. In vivo imaging of gene transfer to the respiratory tract
8. Endostatin gene transfection using a cationic lipid: advantages of transfection before tumor cell inoculation and repeated transfection
9. Limitations of the Murine Nose in the Development of Nonviral Airway Gene Transfer
10. Surfactant Protein-D Inhibits Lung Inflammation Caused by Ventilation in Premature Newborn Lambs
11. Intratracheal Recombinant Surfactant Protein D Prevents Endotoxin Shock in the Newborn Preterm Lamb
12. Transforming growth factor–β1 gene transfer ameliorates acute lung allograft rejection
13. EX VIVO TRANSFECTION OF TRANSFORMING GROWTH FACTOR-β1 GENE TO PULMONARY ARTERY SEGMENTS IN LUNG GRAFTS
14. LIPOSOME-MEDIATED GENE TRANSFER IN RAT LUNG TRANSPLANTATION: A COMPARISON BETWEEN THE IN VIVO AND EX VIVO APPROACHES
15. EX VIVO LIPOSOME-MEDIATED GENE TRANSFER TO LUNG ISOGRAFTS
16. LIPOSOME-MEDIATED GENE TRANSFER TO LUNG ISOGRAFTS
17. SUCCESSFUL IN VIVO AND EX VIVO TRANSFECTION OF PULMONARY ARTERY SEGMENTS IN LUNG ISOGRAFTS
18. Role of viral hemagglutinin glycosylation in anti-influenza activities of recombinant surfactant protein D
19. Inefficient cationic lipid-mediated siRNA and antisense oligonucleotide transfer to airway epithelial cells in vivo
20. Transfection of pulmonary artery segments in lung isografts during storage
21. Ex vivo transfection of pulmonary artery segments in lung isografts
22. 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
23. Antisense Oligonucleotide-mediated Suppression of Muscle Glycogen Synthase 1 Synthesis as an Approach for Substrate Reduction Therapy of Pompe Disease
24. Dysregulation of Multiple Facets of Glycogen Metabolism in a Murine Model of Pompe Disease
25. Systemic Delivery of a Glucosylceramide Synthase Inhibitor Reduces CNS Substrates and Increases Lifespan in a Mouse Model of Type 2 Gaucher Disease
26. Systemic Administration of AAV8-α-galactosidase A Induces Humoral Tolerance in Nonhuman Primates Despite Low Hepatic Expression
27. Iminosugar-Based Inhibitors of Glucosylceramide Synthase Increase Brain Glycosphingolipids and Survival in a Mouse Model of Sandhoff Disease
28. Induction of Immune Tolerance to a Therapeutic Protein by Intrathymic Gene Delivery
29. Substrate Reduction Augments the Efficacy of Enzyme Therapy in a Mouse Model of Fabry Disease
30. Preexisting Immunity and Low Expression in Primates Highlight Translational Challenges for Liver-directed AAV8-mediated Gene Therapy
31. Evaluation of Systemic Follistatin as an Adjuvant to Stimulate Muscle Repair and Improve Motor Function in Pompe Mice
32. Low-frequency ultrasound increases non-viral gene transfer to the mouse lung
33. Surfactant protein-D regulates the postnatal maturation of pulmonary surfactant lipid pool sizes
34. Role of viral hemagglutinin glycosylation in anti-influenza activities of recombinant surfactant protein D
35. Systemic Insulin-like Growth Factor-1 Reverses Hypoalgesia and Improves Mobility in a Mouse Model of Diabetic Peripheral Neuropathy
36. 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
37. Timing of Therapeutic Intervention Determines Functional and Survival Outcomes in a Mouse Model of Late Infantile Batten Disease
38. Inefficient cationic lipid-mediated siRNA and antisense oligonucleotide transfer to airway epithelial cells in vivo
39. 475. SeV-Mediated Gene Transfer after Three Re-Administrations in Mice and Sheep
40. 217. Comparison of Hepatic Vein and Peripheral Vein Delivery of an AAV2/8 Vector Encoding Human a-Galactosidase A to Rhesus Macaques
41. 893. Second Generation Gene Therapy Vector for Classical Late Infantile Neuronal Ceroid Lipofuscinosis
42. 695. Low-Frequency Ultrasound Increases Non- Viral Lung Gene Transfer
43. Gene therapy for lung cancer - an application for cationic lipid-mediated gene delivery?
44. Local Delivery of a Viral Vector Mitigates Neutralization by Antiviral Antibodies and Results in Efficient Transduction of Rabbit Liver
45. Transient siRNA-Mediated Attenuation of Liver Expression from an α-Galactosidase a Plasmid Reduces Subsequent Humoral Immune Responses to the Transgene Product in Mice
46. Long-term Transgene Expression from Plasmid DNA Gene Therapy Vectors Is Negatively Affected by CpG Dinucleotides
47. Contribution of Toll-like Receptor 9 Signaling to the Acute Inflammatory Response to Nonviral Vectors
48. DNA Sequences in Cationic Lipid:pDNA-Mediated Systemic Toxicities
49. Development of Catheter-Based Procedures for Transducing the Isolated Rabbit Liver with Plasmid DNA
50. Tumor Treatment with Complexes of Cationic Lipid and Noncoding Plasmid DNA Results in the Induction of Cytotoxic T Cells and Systemic Tumor Elimination
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