29 results on '"Cheng, Seng"'
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2. Gaucher-related synucleinopathies: The examination of sporadic neurodegeneration from a rare (disease) angle
3. GENE THERAPEUTIC STRATEGIES FOR TREATING SPINAL MUSCULAR ATROPHY: S8-5.
4. Reversibility of neuropathology in Tay-Sachs-related diseases
5. A randomised, double-blind, placebo-controlled phase IIB clinical trial of repeated application of gene therapy in patients with cystic fibrosis
6. Targeting nuclear RNA for in vivo correction of myotonic dystrophy
7. Assessment of the nuclear pore dilating agent trans-cyclohexane-1,2-diol in differentiated airway epithelium
8. Marked differences in neurochemistry and aggregates despite similar behavioural and neuropathological features of Huntington disease in the full-length BACHD and YAC128 mice
9. Adeno-associated virus-mediated expression of β-hexosaminidase prevents neuronal loss in the Sandhoff mouse brain
10. Acid β-Glucosidase Mutants Linked To Gaucher Disease, Parkinson Disease, and Lewy Body Dementia Alter α-Synuclein Processing
11. Adeno-associated virus-mediated expression of acid sphingomyelinase decreases atherosclerotic lesion formation in apolipoprotein E−/− mice
12. Relationship between neuropathology and disease progression in the SOD1G93A ALS mouse
13. Inhibition of osteoclastogenesis by prolyl hydroxylase inhibitor dimethyloxallyl glycine
14. Limitations of the Murine Nose in the Development of Nonviral Airway Gene Transfer
15. Reducing glycosphingolipid biosynthesis in airway cells partially ameliorates disease manifestations in a mouse model of asthma
16. Improved management of lysosomal glucosylceramide levels in a mouse model of type 1 Gaucher disease using enzyme and substrate reduction therapy
17. CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy
18. Inhibiting glycosphingolipid synthesis ameliorates hepatic steatosis in obese mice#
19. Intracerebroventricular infusion of acid sphingomyelinase corrects CNS manifestations in a mouse model of Niemann–Pick A disease
20. Temporal Neuropathologic and Behavioral Phenotype of 6neo/6neo Pompe Disease Mice
21. Intraparenchymal injections of acid sphingomyelinase results in regional correction of lysosomal storage pathology in the Niemann–Pick A mouse
22. Acute intermittent porphyria: vector optimization for gene therapy
23. Inhibiting Glycosphingolipid Synthesis Improves Glycemic Control and Insulin Sensitivity in Animal Models of Type 2 Diabetes
24. Electroporation enhances reporter gene expression following delivery of naked plasmid DNA to the lung
25. Inhibiting Glycosphingolipid Synthesis Improves Glycemic Control and Insulin Sensitivity in Animal Models of Type 2 Diabetes: 449-P
26. AAV8-mediated expression of glucocerebrosidase ameliorates the storage pathology in the visceral organs of a mouse model of Gaucher disease
27. Restoration of Cyclic Adenosine Monophosphate-Stimulated Chloride Channel Activity in Human Cystic Fibrosis Tracheobronchial Submucosal Gland Cells by Adenovirus-Mediated and Cationic Lipid-Mediated Gene Transfer
28. Components of Human Papillomavirus That Activate Transcription and Support Plasmid Replication in Human Airway Cells
29. Comparison of DNA-Lipid Complexes and DNA Alone for Gene Transfer to Cystic Fibrosis Airway Epithelia In Vivo
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