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49 results on '"Scheule, Ronald"'

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1. Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial

2. Antisense oligonucleotide-mediated suppression of muscle glycogen synthase 1 synthesis as an approach for substrate reduction therapy of Pompe disease

3. Antisense Oligonucleotide-mediated Suppression of Muscle Glycogen Synthase 1 Synthesis as an Approach for Substrate Reduction Therapy of Pompe Disease

4. The safety profile of a cationic lipid-mediated cystic fibrosis gene transfer agent following repeated monthly aerosol administration to sheep

6. Iminosugar-based inhibitors of glucosylceramide synthase prolong survival but paradoxically increase brain glucosylceramide levels in Niemann–Pick C mice

9. Systemic Administration of AAV8-α-galactosidase A Induces Humoral Tolerance in Nonhuman Primates Despite Low Hepatic Expression

10. Distribution of acid sphingomyelinase in rodent and non-human primate brain after intracerebroventricular infusion

11. Secreted Gaussia luciferase as a sensitive reporter gene for in vivo and ex vivo studies of airway gene transfer

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

14. Preexisting Immunity and Low Expression in Primates Highlight Translational Challenges for Liver-directed AAV8-mediated Gene Therapy

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

17. The use of carboxymethylcellulose gel to increase non-viral gene transfer in mouse airways

20. The role of doxorubicin in non-viral gene transfer in the lung

22. In vivo imaging of gene transfer to the respiratory tract

24. Timing of Therapeutic Intervention Determines Functional and Survival Outcomes in a Mouse Model of Late Infantile Batten Disease

25. 475. SeV-Mediated Gene Transfer after Three Re-Administrations in Mice and Sheep

28. 695. Low-Frequency Ultrasound Increases Non- Viral Lung Gene Transfer

42. Ex vivo liposome-mediated gene transfer to lung isografts

44. Liposome-mediated gene transfer to lung isografts

45. Purification and Characterization of Recombinant Cystic Fibrosis Transmembrane Conductance Regulator from Chinese Hamster Ovary and Insect Cells

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