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

54. Low-frequency ultrasound increases non-viral gene transfer to the mouse lung

55. Detection of CFTR transgene mRNA expression in respiratory epithelium isolated from the murine nasal cavity

59. 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

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

61. Inefficient cationic lipid-mediated siRNA and antisense oligonucleotide transfer to airway epithelial cells in vivo

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

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

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

88. Basis of Pulmonary Toxicity Associated with Cationic Lipid-Mediated Gene Transfer to the Mammalian Lung

89. Detailed Analysis of Structures and Formulations of Cationic Lipids for Efficient Gene Transfer to the Lung

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

94. Detection of CFTR transgene mRNA expression in respiratory epithelium isolated from the murine nasal cavity.

98. Surfactant Protein-D and Surfactant Inhibit Endotoxin-Induced Pulmonary lnflammation.

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