464 results on '"Cheng, Seng"'
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2. Glucosylceramide synthase inhibition reduces ganglioside GM3 accumulation, alleviates amyloid neuropathology, and stabilizes remote contextual memory in a mouse model of Alzheimer’s disease
3. Adeno-associated viral vector serotype 9–based gene therapy for Niemann-Pick disease type A
4. Realizing the promise of gene therapy through collaboration and partnering : Pfizer’s view
5. Molecular characterization of 'OsCURT1A' from upland rice in response to osmotic stress
6. Molecular characterisation of nerol dehydrogenase gene (PmNeDH) from Persicaria minor in response to stress-related phytohormones
7. Relicensing Practices of Taxi Drivers and Crane Operators Aged 70 Years and above in Singapore
8. Merits of Combination Cortical, Subcortical, and Cerebellar Injections for the Treatment of Niemann-Pick Disease Type A
9. Fetal gene therapy for neurodegenerative disease of infants
10. 'Benign' temporal lobe epilepsy with hippocampal sclerosis: A forgotten entity?
11. Gene Transfer of Human Acid Sphingomyelinase Corrects Neuropathology and Motor Deficits in a Mouse Model of Niemann-Pick Type A Disease
12. Glucosylceramide synthase inhibition alleviates aberrations in synucleinopathy models
13. Microarray dataset of transgenic rice overexpressing Abp57
14. Intravitreous injection of AAV2-sFLT01 in patients with advanced neovascular age-related macular degeneration: a phase 1, open-label trial
15. Rationally designed AAV2 and AAVrh8R capsids provide improved transduction in the retina and brain
16. Priapism in a Fabry disease mouse model is associated with upregulated penile nNOS and eNOS expression
17. A Novel Cationic Lipid Greatly Enhances Plasmid DNA Delivery and Expression in Mouse Lung
18. Agarwood Induction: Current Developments and Future Perspectives
19. α-Galactosidase A Knockout Mice: Progressive Organ Pathology Resembles the Type 2 Later-Onset Phenotype of Fabry Disease
20. Glycosphingolipids are modulators of disease pathogenesis in amyotrophic lateral sclerosis
21. The safety profile of a cationic lipid-mediated cystic fibrosis gene transfer agent following repeated monthly aerosol administration to sheep
22. cDNA cloning and expression analysis of the chalcone synthase gene (CHS) from 'polygonum minus
23. Metabolic signatures of amyotrophic lateral sclerosis reveal insights into disease pathogenesis
24. Augmenting CNS glucocerebrosidase activity as a therapeutic strategy for parkinsonism and other Gaucher-related synucleinopathies
25. Tetrahydrobiopterin deficiency in the pathogenesis of Fabry disease
26. Efficacy of Enzyme and Substrate Reduction Therapy with a Novel Antagonist of Glucosylceramide Synthase for Fabry Disease
27. IGF-1 delivery to CNS attenuates motor neuron cell death but does not improve motor function in type III SMA mice
28. CNS expression of glucocerebrosidase corrects α-synuclein pathology and memory in a mouse model of Gaucher-related synucleinopathy
29. Secreted Gaussia luciferase as a sensitive reporter gene for in vivo and ex vivo studies of airway gene transfer
30. Prospects for the gene therapy of spinal muscular atrophy
31. Relationship between neuropathology and disease progression in the SOD1 G93A ALS mouse
32. Inhibiting TGF-β Activity Improves Respiratory Function in mdx Mice
33. Combination Brain and Systemic Injections of AAV Provide Maximal Functional and Survival Benefits in the Niemann-Pick Mouse
34. Effective Gene Therapy in an Authentic Model of Tay-Sachs-Related Diseases
35. The use of carboxymethylcellulose gel to increase non-viral gene transfer in mouse airways
36. Gaucher-related synucleinopathies: The examination of sporadic neurodegeneration from a rare (disease) angle
37. The role of doxorubicin in non-viral gene transfer in the lung
38. Intracerebroventricular infusion of acid sphingomyelinase corrects CNS manifestations in a mouse model of Niemann–Pick A disease
39. Rapid identification of novel functional promoters for gene therapy
40. Targeting nuclear RNA for in vivo correction of myotonic dystrophy
41. Neural Stem Cell Transplantation as a Therapeutic Approach for Treating Lysosomal Storage Diseases
42. In vivo imaging of gene transfer to the respiratory tract
43. Inhibition of osteoclastogenesis by prolyl hydroxylase inhibitor dimethyloxallyl glycine
44. Improved management of lysosomal glucosylceramide levels in a mouse model of type 1 Gaucher disease using enzyme and substrate reduction therapy
45. CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy
46. GENE THERAPEUTIC STRATEGIES FOR TREATING SPINAL MUSCULAR ATROPHY: S8-5.
47. Reversibility of neuropathology in Tay-Sachs-related diseases
48. “Benign” temporal lobe epilepsy with hippocampal sclerosis: A forgotten entity?
49. AAV‐mediated expression of galactose‐1‐phosphate uridyltransferase corrects defects of galactose metabolism in classic galactosemia patient fibroblasts.
50. A randomised, double-blind, placebo-controlled phase IIB clinical trial of repeated application of gene therapy in patients with cystic fibrosis
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