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

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

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

212. Glycoengineered Acid α-Glucosidase With Improved Efficacy at Correcting the Metabolic Aberrations and Motor Function Deficits in a Mouse Model of Pompe Disease

214. Pulmonary delivery of recombinant acid sphingomyelinase improves clearance of lysosomal sphingomyelin from the lungs of a murine model of Niemann–Pick disease

215. Optical Signal Modulation of Semiconductor Soliton Device with Circular Ring Resonator

217. 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

218. Delivery of AAV-IGF-1 to the CNS Extends Survival in ALS Mice Through Modification of Aberrant Glial Cell Activity

219. CpG-free plasmids confer reduced inflammation and sustained pulmonary gene expression

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

223. A specific and potent inhibitor of glucosylceramide synthase for substrate inhibition therapy of Gaucher disease

226. Optimizing Aerosol Gene Delivery and Expression in the Ovine Lung

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

230. Intracranial Delivery of CLN2 Reduces Brain Pathology in a Mouse Model of Classical Late Infantile Neuronal Ceroid Lipofuscinosis

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

232. 2. Identification of Different Modes of Viral Transport in the Non-Human Primate Brain after Convection-Enhanced Delivery of AAV Serotype Vectors

239. 1062. Intracerebellar Injection of AAV-IGF-1 Improves Motor Function and Extends Survival in a Mouse Model of Amyotrophic Lateral Sclerosis

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

243. Adenovirus-Mediated Expression of β-Adrenergic Receptor Kinase C-Terminus Reduces Intimal Hyperplasia and Luminal Stenosis of Arteriovenous Polytetrafluoroethylene Grafts in Pigs

248. Effect of Baseline or Changes in Adrenergic Activity on Clinical Outcomes in the β-Blocker Evaluation of Survival Trial

249. Catheter‐mediated delivery of adenoviral vectors expressing β‐adrenergic receptor kinase C‐terminus inhibits intimal hyperplasia and luminal stenosis in rabbit iliac arteries

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