809 results on '"Cheng, Seng"'
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2. Adeno-associated viral vector serotype 9–based gene therapy for Niemann-Pick disease type A
3. Glucosylceramide synthase inhibition reduces ganglioside GM3 accumulation, alleviates amyloid neuropathology, and stabilizes remote contextual memory in a mouse model of Alzheimer’s disease
4. A plain language summary of AAV-based gene therapy: challenges and potential solutions for people with antibodies against AAV
5. Rationale for using centralized transduction inhibition assays in three phase 3 rAAV gene therapy clinical trials
6. Realizing the promise of gene therapy through collaboration and partnering : Pfizer’s view
7. Liposome delivery systems
8. Molecular characterisation of nerol dehydrogenase gene (PmNeDH) from Persicaria minor in response to stress-related phytohormones
9. Relicensing Practices of Taxi Drivers and Crane Operators Aged 70 Years and above in Singapore
10. Merits of Combination Cortical, Subcortical, and Cerebellar Injections for the Treatment of Niemann-Pick Disease Type A
11. Fetal gene therapy for neurodegenerative disease of infants
12. 'Benign' temporal lobe epilepsy with hippocampal sclerosis: A forgotten entity?
13. Gene Transfer of Human Acid Sphingomyelinase Corrects Neuropathology and Motor Deficits in a Mouse Model of Niemann-Pick Type A Disease
14. Glucosylceramide synthase inhibition alleviates aberrations in synucleinopathy models
15. Microarray dataset of transgenic rice overexpressing Abp57
16. Intravitreous injection of AAV2-sFLT01 in patients with advanced neovascular age-related macular degeneration: a phase 1, open-label trial
17. GENE THERAPY OF NEUROLOGICAL DISEASES
18. Rationally designed AAV2 and AAVrh8R capsids provide improved transduction in the retina and brain
19. Priapism in a Fabry disease mouse model is associated with upregulated penile nNOS and eNOS expression
20. Dysregulated DNA methylation in the pathogenesis of Fabry disease
21. A Novel Cationic Lipid Greatly Enhances Plasmid DNA Delivery and Expression in Mouse Lung
22. Agarwood Induction: Current Developments and Future Perspectives
23. α-Galactosidase A Knockout Mice: Progressive Organ Pathology Resembles the Type 2 Later-Onset Phenotype of Fabry Disease
24. Glycosphingolipids are modulators of disease pathogenesis in amyotrophic lateral sclerosis
25. Adeno-Associated Viral-Mediated Gene Therapy of Lysosomal Storage Disorders
26. The safety profile of a cationic lipid-mediated cystic fibrosis gene transfer agent following repeated monthly aerosol administration to sheep
27. Metabolic signatures of amyotrophic lateral sclerosis reveal insights into disease pathogenesis
28. Augmenting CNS glucocerebrosidase activity as a therapeutic strategy for parkinsonism and other Gaucher-related synucleinopathies
29. Additional file 5 of Glucosylceramide synthase inhibition reduces ganglioside GM3 accumulation, alleviates amyloid neuropathology, and stabilizes remote contextual memory in a mouse model of Alzheimer���s disease
30. Tetrahydrobiopterin deficiency in the pathogenesis of Fabry disease
31. Design of a dynamic fuzzy controller IC with application to garage parking control.
32. Efficacy of Enzyme and Substrate Reduction Therapy with a Novel Antagonist of Glucosylceramide Synthase for Fabry Disease
33. IGF-1 delivery to CNS attenuates motor neuron cell death but does not improve motor function in type III SMA mice
34. AAV ‐mediated expression of galactose‐1‐phosphate uridyltransferase corrects defects of galactose metabolism in classic galactosemia patient fibroblasts
35. CNS expression of glucocerebrosidase corrects α-synuclein pathology and memory in a mouse model of Gaucher-related synucleinopathy
36. Molecular characterization of OsCURT1A from upland rice in response to osmotic stress
37. Secreted Gaussia luciferase as a sensitive reporter gene for in vivo and ex vivo studies of airway gene transfer
38. Prospects for the gene therapy of spinal muscular atrophy
39. Relationship between neuropathology and disease progression in the SOD1 G93A ALS mouse
40. Inhibiting TGF-β Activity Improves Respiratory Function in mdx Mice
41. Combination Brain and Systemic Injections of AAV Provide Maximal Functional and Survival Benefits in the Niemann-Pick Mouse
42. Effective Gene Therapy in an Authentic Model of Tay-Sachs-Related Diseases
43. Function and Regulation of the Cystic Fibrosis Transmembrane Conductance Regulator
44. The use of carboxymethylcellulose gel to increase non-viral gene transfer in mouse airways
45. Gaucher-related synucleinopathies: The examination of sporadic neurodegeneration from a rare (disease) angle
46. The role of doxorubicin in non-viral gene transfer in the lung
47. Intracerebroventricular infusion of acid sphingomyelinase corrects CNS manifestations in a mouse model of Niemann–Pick A disease
48. Rapid identification of novel functional promoters for gene therapy
49. 23: GENE THERAPY OF NEUROLOGICAL DISEASES
50. Targeting nuclear RNA for in vivo correction of myotonic dystrophy
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