256 results on '"Cheng, Seng"'
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2. Rationale for using centralized transduction inhibition assays in three phase 3 rAAV gene therapy clinical trials
3. Liposome delivery systems
4. GENE THERAPY OF NEUROLOGICAL DISEASES
5. Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy
6. Dysregulated DNA methylation in the pathogenesis of Fabry disease
7. Glucosylceramide synthase inhibition reduces ganglioside GM3 accumulation, alleviates amyloid neuropathology, and stabilizes remote contextual memory in a mouse model of Alzheimer’s disease
8. AAV ‐mediated expression of galactose‐1‐phosphate uridyltransferase corrects defects of galactose metabolism in classic galactosemia patient fibroblasts
9. 23: GENE THERAPY OF NEUROLOGICAL DISEASES
10. Therapies for Lysosomal Storage Diseases
11. Substrate reduction therapy using Genz‐667161 reduces levels of pathogenic components in a mouse model of neuronopathic forms of Gaucher disease
12. biAb Mediated Restoration of the Linkage between Dystroglycan and Laminin-211 as a Therapeutic Approach for α-Dystroglycanopathies
13. “Benign” temporal lobe epilepsy with hippocampal sclerosis: A forgotten entity?
14. Adeno-Associated Viral-Mediated Gene Therapy of Lysosomal Storage Disorders
15. Prospects for Clinical Application of Synthetic Gene Delivery Vectors
16. Sustaining Transgene Expression in Vivo
17. Cationic Lipid-Mediated Gene Therapy
18. Cationic Lipid-Mediated Gene Delivery to the Airways
19. [52] Cationic lipid formulations for intracellular gene delivery of cystic fibrosis transmembrane conductance regulator to airway epithelia
20. Adeno-associated viral vector serotype 9–based gene therapy for Niemann-Pick disease type A
21. Molecular characterization of OsCURT1A from upland rice in response to osmotic stress
22. Substrate Reduction Therapy for Sandhoff Disease through Inhibition of Glucosylceramide Synthase Activity
23. Agarwood Induction: Current Developments and Future Perspectives
24. Molecular characterisation of nerol dehydrogenase gene (PmNeDH) from Persicaria minor in response to stress-related phytohormones
25. Function and Regulation of the Cystic Fibrosis Transmembrane Conductance Regulator
26. Inhibiting neutral amino acid transport for the treatment of phenylketonuria
27. Fetal gene therapy for neurodegenerative disease of infants
28. Universal Method for the Purification of Recombinant AAV Vectors of Differing Serotypes
29. Rationally designed AAV2 and AAVrh8R capsids provide improved transduction in the retina and brain
30. Efficacy of Genz-682452-mediated inhibition of glucosylceramide synthase in a mouse model of Sandhoff disease
31. Priapism in a Fabry disease mouse model is associated with upregulated penile nNOS and eNOS expression
32. Structural and kinetic studies of a novel nerol dehydrogenase from Persicaria minor, a nerol-specific enzyme for citral biosynthesis
33. Priapism in a Fabry disease mouse model is associated with upregulated penile nNOS and eNOS expression
34. Microarray dataset of transgenic rice overexpressing Abp57
35. AB008. Bifunctional antibody as a surrogate molecular linker for the treatment of alpha-dystroglycan related muscular dystrophies
36. Intravitreous injection of AAV2-sFLT01 in patients with advanced neovascular age-related macular degeneration: a phase 1, open-label trial
37. AAVS1-Targeted Plasmid Integration in AAV Producer Cell Lines
38. Glucosylceramide synthase inhibition alleviates aberrations in synucleinopathy models
39. Tetrahydrobiopterin deficiency in the pathogenesis of Fabry disease
40. Characteristics of Minimally Oversized Adeno-Associated Virus Vectors Encoding Human Factor VIII Generated Using Producer Cell Lines and Triple Transfection
41. CRISPR/Cas9-Mediated Genome Editing as a Therapeutic Approach for Leber Congenital Amaurosis 10
42. Crystallization and X-ray crystallographic analysis of recombinant TylP, a putative γ-butyrolactone receptor protein fromStreptomyces fradiae
43. Tetrahydrobiopterin deficiency in the pathogenesis of Fabry disease
44. CNS-accessible Inhibitor of Glucosylceramide Synthase for Substrate Reduction Therapy of Neuronopathic Gaucher Disease
45. 621. Widespread Gene Delivery to the Nonhuman Primate Brain for the Treatment of Huntington's Disease
46. 92. Evaluation of Producer Cell Line Platform for Production of Oversized AAV-FVIII Vectors
47. 301. AAV Capsid Engineering to Improve Transduction in Retina and Brain
48. 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
49. 329. Using CRISPR/Cas9 as a Therapeutic Approach for Leber Congenital Amaurosis 10 (LCA10)
50. Glucocerebrosidase modulates cognitive and motor activities in murine models of Parkinson’s disease
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