250 results on '"Boye, Shannon E."'
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2. Engineered AAV capsid transport mutants overcome transduction deficiencies in the aged CNS
3. Optimization of Capillary-Based Western Blotting for MYO7A
4. Intravitreal injection of a rationally designed AAV capsid library in non-human primate identifies variants with enhanced retinal transduction and neutralizing antibody evasion
5. Dual-AAV vector-mediated expression of MYO7A improves vestibular function in a mouse model of Usher syndrome 1B
6. Development of an AAV-CRISPR-Cas9-based treatment for dominant cone-rod dystrophy 6
7. Preclinical studies in support of phase I/II clinical trials to treat GUCY2D-associated Leber congenital amaurosis
8. Post-developmental plasticity of the primary rod pathway allows restoration of visually guided behaviors
9. Night vision restored in days after decades of congenital blindness
10. Correction: Site-specific modifications to AAV8 capsid yields enhanced brain transduction in the neonatal MPS IIIB mouse
11. Safety and improved efficacy signals following gene therapy in childhood blindness caused by GUCY2D mutations
12. Site-specific modifications to AAV8 capsid yields enhanced brain transduction in the neonatal MPS IIIB mouse
13. Current Clinical Applications of In Vivo Gene Therapy with AAVs
14. Promotion of endoplasmic reticulum retrotranslocation by overexpression of E3 ubiquitin‐protein ligase synoviolin 1 reduces endoplasmic reticulum stress and preserves cone photoreceptors in cyclic nucleotide‐gated channel deficiency.
15. Novel AAV44.9-Based Vectors Display Exceptional Characteristics for Retinal Gene Therapy
16. Safety and improved efficacy signals following gene therapy in childhood blindness caused by GUCY2D mutations
17. Safety and efficacy of ATSN-101 in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D: a phase 1/2, multicentre, open-label, unilateral dose escalation study
18. Gene therapy: charting a future course--summary of a National Institutes of Health Workshop, April 12, 2013.
19. A Drug-Tunable Gene Therapy for Broad-Spectrum Protection against Retinal Degeneration
20. A Mini-review: Animal Models of GUCY2D Leber Congenital Amaurosis (LCA1)
21. Rationally Engineered AAV Capsids Improve Transduction and Volumetric Spread in the CNS
22. Optimization of Retinal Gene Therapy for X-Linked Retinitis Pigmentosa Due to RPGR Mutations
23. Defining Outcomes for Clinical Trials of Leber Congenital Amaurosis Caused by GUCY2D Mutations
24. Utilizing minimally purified secreted rAAV for rapid and cost-effective manipulation of gene expression in the CNS
25. SubILM Injection of AAV for Gene Delivery to the Retina
26. Improving retinal vascular endothelial cell tropism through rational rAAV capsid design
27. NF1 Is a Direct G Protein Effector Essential for Opioid Signaling to Ras in the Striatum
28. Cone Specific Promoter for Use in Gene Therapy of Retinal Degenerative Diseases
29. Identifying and Overcoming Challenges in Developing Effective Treatments for Usher 1B: A Workshop Report
30. Gene Therapy With the Caspase Activation and Recruitment Domain Reduces the Ocular Inflammatory Response
31. Targeted Gene Delivery to the Enteric Nervous System Using AAV: A Comparison Across Serotypes and Capsid Mutants
32. Targeted CNS delivery using human MiniPromoters and demonstrated compatibility with adeno-associated viral vectors
33. Gene Therapy in Opn1mw−/−/Opn1sw−/− Mice and Implications for Blue Cone Monochromacy Patients with Deletion Mutations
34. A Comprehensive Review of Retinal Gene Therapy
35. Night Vision Restored in Days After Decades of Congenital Blindness
36. Gene delivery to mitochondria by targeting modified adenoassociated virus suppresses Leber's hereditary optic neuropathy in a mouse model
37. Long-term RNA interference gene therapy in a dominant retinitis pigmentosa mouse model
38. Virally delivered Channelrhodopsin-2 Safely and Effectively Restores Visual Function in Multiple Mouse Models of Blindness
39. Long-term Retinal Function and Structure Rescue Using Capsid Mutant AAV8 Vector in the rd10 Mouse, a Model of Recessive Retinitis Pigmentosa
40. A Mini-review: Animal Models of GUCY2D Leber Congenital Amaurosis (LCA1)
41. Intravitreal delivery of a novel AAV vector targets ON bipolar cells and restores visual function in a mouse model of complete congenital stationary night blindness
42. Cone Specific Promoter for Use in Gene Therapy of Retinal Degenerative Diseases
43. Gene Therapy in Opn1mw−/−/Opn1sw−/− Mice and Implications for Blue Cone Monochromacy Patients with Deletion Mutations.
44. Effects of Altering Heparan Sulfate Proteoglycan Binding and Capsid Hydrophilicity on Retinal Transduction by Adeno-associated Virus
45. Retinal Diseases
46. Site-specific modifications to AAV8 capsid yields enhanced brain transduction in the neonatal MPS IIIB mouse
47. SARM1 depletion rescues NMNAT1-dependent photoreceptor cell death and retinal degeneration
48. Author response: SARM1 depletion rescues NMNAT1-dependent photoreceptor cell death and retinal degeneration
49. Identifying Treatments for Taste and Smell Disorders: Gaps and Opportunities
50. Adeno-Associated Virus D-Sequence-Mediated Suppression of Expression of a Human Major Histocompatibility Class II Gene: Implications in the Development of Adeno-Associated Virus Vectors for Modulating Humoral Immune Response
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