114 results on '"Cheng, Seng"'
Search Results
2. Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy
3. Dysregulated DNA methylation in the pathogenesis of Fabry disease
4. Structural and kinetic studies of a novel nerol dehydrogenase from Persicaria minor, a nerol-specific enzyme for citral biosynthesis
5. biAb Mediated Restoration of the Linkage between Dystroglycan and Laminin-211 as a Therapeutic Approach for α-Dystroglycanopathies
6. “Benign” temporal lobe epilepsy with hippocampal sclerosis: A forgotten entity?
7. Substrate Reduction Therapy for Sandhoff Disease through Inhibition of Glucosylceramide Synthase Activity
8. Universal Method for the Purification of Recombinant AAV Vectors of Differing Serotypes
9. Efficacy of Genz-682452-mediated inhibition of glucosylceramide synthase in a mouse model of Sandhoff disease
10. Priapism in a Fabry disease mouse model is associated with upregulated penile nNOS and eNOS expression
11. Structural and kinetic studies of a novel nerol dehydrogenase from Persicaria minor, a nerol-specific enzyme for citral biosynthesis
12. Microarray dataset of transgenic rice overexpressing Abp57
13. Intravitreous injection of AAV2-sFLT01 in patients with advanced neovascular age-related macular degeneration: a phase 1, open-label trial
14. CRISPR/Cas9-Mediated Genome Editing as a Therapeutic Approach for Leber Congenital Amaurosis 10
15. Tetrahydrobiopterin deficiency in the pathogenesis of Fabry disease
16. CNS-accessible Inhibitor of Glucosylceramide Synthase for Substrate Reduction Therapy of Neuronopathic Gaucher Disease
17. 621. Widespread Gene Delivery to the Nonhuman Primate Brain for the Treatment of Huntington's Disease
18. 92. Evaluation of Producer Cell Line Platform for Production of Oversized AAV-FVIII Vectors
19. 301. AAV Capsid Engineering to Improve Transduction in Retina and Brain
20. 249. Preliminary Results of a Phase 1, Open-Label, Safety and Tolerability Study of a Single Intravitreal Injection of AAV2-sFLT01 in Patients with Neovascular Age-Related Macular Degeneration
21. 329. Using CRISPR/Cas9 as a Therapeutic Approach for Leber Congenital Amaurosis 10 (LCA10)
22. AAV8-mediated expression of N-acetylglucosamine-1-phosphate transferase attenuates bone loss in a mouse model of mucolipidosis II
23. Glucosylceramide synthase inhibition reduces α-synuclein pathology and improves cognition in murine models of synucleinopathy
24. The impact of minimally oversized adeno-associated viral vectors encoding human factor VIII on vector potency in vivo
25. Widespread AAV1- and AAV2-mediated transgene expression in the nonhuman primate brain: implications for Huntington's disease
26. Genetic variation in the mitochondrial genome of the giant grouper Epinephelus lanceolatus (Bloch, 1790) and its application for the identification of broodstock
27. Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial
28. 247. Characterization of Oversized rAAV Vectors for Human FVIII Gene Transfer
29. 556. Analytical Ultracentrifugation as an Approach to Characterize Recombinant AAV Vectors
30. α-Galactosidase A Knockout Mice
31. Gaucher-related synucleinopathies: The examination of sporadic neurodegeneration from a rare (disease) angle
32. Antisense oligonucleotide-mediated suppression of muscle glycogen synthase 1 synthesis as an approach for substrate reduction therapy of Pompe disease
33. Evaluation of a novel substrate reduction therapy with CNS access in mouse models of neuronopathic Gaucher disease
34. Gene therapy for the neurological manifestations in lysosomal storage disorders
35. Systemic Administration of a Recombinant AAV1 Vector Encoding IGF-1 Improves Disease Manifestations in SMA Mice
36. Antisense Oligonucleotide-mediated Suppression of Muscle Glycogen Synthase 1 Synthesis as an Approach for Substrate Reduction Therapy of Pompe Disease
37. The safety profile of a cationic lipid-mediated cystic fibrosis gene transfer agent following repeated monthly aerosol administration to sheep
38. Erythrocytes encapsulated with phenylalanine hydroxylase exhibit improved pharmacokinetics and lowered plasma phenylalanine levels in normal mice
39. Therapeutic Response in Feline Sandhoff Disease Despite Immunity to Intracranial Gene Therapy
40. Augmenting glucocerebrosidase activity in the CNS as a therapeutic strategy for Gaucher-related synucleinopathies
41. A novel, selective and orally-available glucosylceramide synthase inhibitor for substrate reduction therapy of Fabry disease
42. Analytical performance monitoring of a 142.5kWp grid-connected rooftop BIPV system in Singapore
43. Merits of Combination Cortical, Subcortical, and Cerebellar Injections for the Treatment of Niemann-Pick Disease Type A
44. Gene Transfer to the CNS Is Efficacious in Immune-primed Mice Harboring Physiologically Relevant Titers of Anti-AAV Antibodies
45. Gene Transfer Corrects Acute GM2 Gangliosidosis—Potential Therapeutic Contribution of Perivascular Enzyme Flow
46. Sustained Therapeutic Reversal of Huntington's Disease by Transient Repression of Huntingtin Synthesis
47. Iminosugar-based inhibitors of glucosylceramide synthase prolong survival but paradoxically increase brain glucosylceramide levels in Niemann–Pick C mice
48. Ligation of Cytotoxic T Lymphocyte Antigen-4 to T Cell Receptor Inhibits T Cell Activation and Directs Differentiation into Foxp3+ Regulatory T Cells
49. Glucosylceramide Synthase Inhibition Reduces Gb3 and Lyso-Gb3 in a Mouse Model Of Fabry Disease
50. IGF-1 delivery to CNS attenuates motor neuron cell death but does not improve motor function in type III SMA mice
Catalog
Books, media, physical & digital resources
Discovery Service for Jio Institute Digital Library
For full access to our library's resources, please sign in.