97 results on '"Cheng, Seng"'
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2. Merits of Combination Cortical, Subcortical, and Cerebellar Injections for the Treatment of Niemann-Pick Disease Type A
3. biAb Mediated Restoration of the Linkage between Dystroglycan and Laminin-211 as a Therapeutic Approach for α-Dystroglycanopathies
4. Substrate Reduction Therapy for Sandhoff Disease through Inhibition of Glucosylceramide Synthase Activity
5. CRISPR/Cas9-Mediated Genome Editing as a Therapeutic Approach for Leber Congenital Amaurosis 10
6. CNS-accessible Inhibitor of Glucosylceramide Synthase for Substrate Reduction Therapy of Neuronopathic Gaucher Disease
7. 621. Widespread Gene Delivery to the Nonhuman Primate Brain for the Treatment of Huntington's Disease
8. 92. Evaluation of Producer Cell Line Platform for Production of Oversized AAV-FVIII Vectors
9. 301. AAV Capsid Engineering to Improve Transduction in Retina and Brain
10. 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
11. 329. Using CRISPR/Cas9 as a Therapeutic Approach for Leber Congenital Amaurosis 10 (LCA10)
12. 247. Characterization of Oversized rAAV Vectors for Human FVIII Gene Transfer
13. 556. Analytical Ultracentrifugation as an Approach to Characterize Recombinant AAV Vectors
14. Systemic Administration of a Recombinant AAV1 Vector Encoding IGF-1 Improves Disease Manifestations in SMA Mice
15. Therapeutic Response in Feline Sandhoff Disease Despite Immunity to Intracranial Gene Therapy
16. Gene Transfer to the CNS Is Efficacious in Immune-primed Mice Harboring Physiologically Relevant Titers of Anti-AAV Antibodies
17. Gene Transfer Corrects Acute GM2 Gangliosidosis—Potential Therapeutic Contribution of Perivascular Enzyme Flow
18. Systemic Administration of AAV8-α-galactosidase A Induces Humoral Tolerance in Nonhuman Primates Despite Low Hepatic Expression
19. Induction of Immune Tolerance to a Therapeutic Protein by Intrathymic Gene Delivery
20. AAV4-mediated Expression of IGF-1 and VEGF Within Cellular Components of the Ventricular System Improves Survival Outcome in Familial ALS Mice
21. Preexisting Immunity and Low Expression in Primates Highlight Translational Challenges for Liver-directed AAV8-mediated Gene Therapy
22. Evaluation of Systemic Follistatin as an Adjuvant to Stimulate Muscle Repair and Improve Motor Function in Pompe Mice
23. AAV8-mediated Gene Therapy Prevents Induced Biochemical Attacks of Acute Intermittent Porphyria and Improves Neuromotor Function
24. Glycoengineered Acid α-Glucosidase With Improved Efficacy at Correcting the Metabolic Aberrations and Motor Function Deficits in a Mouse Model of Pompe Disease
25. Delivery of AAV-IGF-1 to the CNS Extends Survival in ALS Mice Through Modification of Aberrant Glial Cell Activity
26. Intraventricular Enzyme Replacement Improves Disease Phenotypes in a Mouse Model of Late Infantile Neuronal Ceroid Lipofuscinosis
27. Timing of Therapeutic Intervention Determines Functional and Survival Outcomes in a Mouse Model of Late Infantile Batten Disease
28. Correction of the Biochemical and Functional Deficits in Fabry Mice Following AAV8–mediated Hepatic Expression of α-galactosidase A
29. Optimizing Aerosol Gene Delivery and Expression in the Ovine Lung
30. 475. SeV-Mediated Gene Transfer after Three Re-Administrations in Mice and Sheep
31. 2. Identification of Different Modes of Viral Transport in the Non-Human Primate Brain after Convection-Enhanced Delivery of AAV Serotype Vectors
32. 696. Development of Lipid/Peptide (Lip/Tide) Vectors for Respiratory Gene Transfer
33. 786. Development of Zero-CpG Plasmids with Reduced Inflammatory Responses Following Delivery of Lipid/pDNA Complexes to the Mouse Lung
34. 226. Effective Gene Therapy in an Authentic Mouse Model of Tay-Sachs Related Diseases
35. 218. Gene Therapy and Enzyme Replacement in a Mouse Model of Late Infantile Neuronal Ceroid Lipofuscinosis
36. 217. Comparison of Hepatic Vein and Peripheral Vein Delivery of an AAV2/8 Vector Encoding Human a-Galactosidase A to Rhesus Macaques
37. 893. Second Generation Gene Therapy Vector for Classical Late Infantile Neuronal Ceroid Lipofuscinosis
38. 1062. Intracerebellar Injection of AAV-IGF-1 Improves Motor Function and Extends Survival in a Mouse Model of Amyotrophic Lateral Sclerosis
39. 695. Low-Frequency Ultrasound Increases Non- Viral Lung Gene Transfer
40. AAV8-Mediated Hepatic Expression of Acid Sphingomyelinase Corrects the Metabolic Defect in the Visceral Organs of a Mouse Model of Niemann–Pick Disease
41. AAV2 Vector Harboring a Liver-Restricted Promoter Facilitates Sustained Expression of Therapeutic Levels of α-Galactosidase A and the Induction of Immune Tolerance in Fabry Mice
42. Contribution of Toll-like Receptor 9 Signaling to the Acute Inflammatory Response to Nonviral Vectors
43. Transfection efficiency and toxicity following delivery of naked plasmid DNA and cationic lipid–DNA complexes to ovine lung segments
44. Demonstration of Feasibility of In Vivo Gene Therapy for Gaucher Disease Using a Chemically Induced Mouse Model
45. Adenovirus-Transduced Lung as a Portal for Delivering α-Galactosidase A into Systemic Circulation for Fabry Disease
46. CpG-Depleted Plasmid DNA Vectors with Enhanced Safety and Long-Term Gene Expression in Vivo
47. High and Sustained Transgene Expression in Vivo from Plasmid Vectors Containing a Hybrid Ubiquitin Promoter
48. Reduced Inflammatory Response to Plasmid DNA Vectors by Elimination and Inhibition of Immunostimulatory CpG Motifs
49. Induction of Immune Tolerance to a Therapeutic Protein by Intrathymic Gene Delivery.
50. Glycoengineered Acid α-Glucosidase With Improved Efficacy at Correcting the Metabolic Aberrations and Motor Function Deficits in a Mouse Model of Pompe Disease.
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