Effect of gene therapy on visual function in Leber’s congenital amaurosis

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3. Advances in gene therapy technologies to treat retinitis pigmentosa
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6. 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
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7. How genetic errors in GPCRs affect their function: Possible therapeutic strategies
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8. Genotype-Phenotype Correlation for Leber Congenital Amaurosis in Northern Pakistan
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9. Applying Hsp104 to protein-misfolding disordersThis paper is one of a selection of papers published in this special issue entitled 8th International Conference on AAA Proteins and has undergone the Journal's usual peer review process.
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11. Gene therapy for Stargardt disease associated with ABCA4 gene
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12. Leber congenital amaurosis/early-onset severe retinal dystrophy: current management and clinical trials
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13. Detecting Changes in Retinal Function: Analysis with Non-Stationary Weibull Error Regression and Spatial Enhancement (ANSWERS)
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14. Gene therapy in the second eye of RPE65-deficient dogs improves retinal function
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15. An experimental platform for systemic drug delivery to the retina
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16. Functional Outcome in Subretinal Electronic Implants Depends on Foveal Eccentricity
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17. Zinc‐finger‐based transcriptional repression of rhodopsin in a model of dominant retinitis pigmentosa
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18. The Phenotype of Severe Early Childhood Onset Retinal Dystrophy (SECORD) from Mutation of RPE65 and Differentiation from Leber Congenital Amaurosis
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19. Proof of concept for AAV2/5-mediated gene therapy in iPSC-derived retinal pigment epithelium of a choroideremia patient
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20. Functional and Anatomic Consequences of Subretinal Dosing in the Cynomolgus Macaque
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21. Long-Term Preservation of Cones and Improvement in Visual Function Following Gene Therapy in a Mouse Model of Leber Congenital Amaurosis Caused by Guanylate Cyclase-1 Deficiency
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24. Safety and Proof-of-Concept Study of Oral QLT091001 in Retinitis Pigmentosa Due to Inherited Deficiencies of Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT)
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25. Insight into the mechanisms of enhanced retinal transduction by the engineered AAV2 capsid variant -7m8
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26. Adeno-associated Virus: A Key to the Human Genome?
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27. Ocular Delivery of Compacted DNA-Nanoparticles Does Not Elicit Toxicity in the Mouse Retina
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28. Advances in AAV Vector Development for Gene Therapy in the Retina
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29. Ocular gene therapy: current progress and future prospects
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30. Multifunctional nanocomplexes for gene transfer and gene therapy
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31. A review of therapeutic prospects of non-viral gene therapy in the retinal pigment epithelium
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32. A long-term efficacy study of gene replacement therapy for RPGR-associated retinal degeneration
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33. Successful attenuation of humoral immunity to viral capsid and transgenic protein following AAV-mediated gene transfer with a non-depleting CD4 antibody and cyclosporine
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34. Self-complementary AAV-mediated gene therapy restores cone function and prevents cone degeneration in two models of Rpe65 deficiency
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35. Nonsyndromic Retinitis Pigmentosa Overview
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36. Protrudin functions from the endoplasmic reticulum to support axon regeneration in the adult CNS
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37. Adeno-Associated Viruses (AAV) and Host Immunity – A Race Between the Hare and the Hedgehog
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38. Retinal gene therapy
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39. Telomerase gene therapy ameliorates the effects of neurodegeneration associated to short telomeres in mice
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40. The next step in gene delivery: Molecular engineering of adeno-associated virus serotypes
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41. Adeno-Associated Virus Serotype 1 (AAV1)- and AAV5-Antibody Complex Structures Reveal Evolutionary Commonalities in Parvovirus Antigenic Reactivity
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42. Therapeutic Challenges to Retinitis Pigmentosa: From Neuroprotection to Gene Therapy
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43. The retinal phenotype of Usher syndrome: Pathophysiological insights from animal models
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45. Nr2e3 is a genetic modifier that rescues retinal degeneration and promotes homeostasis in multiple models of retinitis pigmentosa
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46. Rescuing the Failing Heart by Targeted Gene Transfer
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47. Retinal gene delivery by adeno-associated virus (AAV) vectors: Strategies and applications
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48. The effect of human gene therapy for RPE65-associated Leber’s congenital amaurosis on visual function: a systematic review and meta-analysis
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49. Technical brief: subretinal injection and electroporation into adult mouse eyes.
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50. AAV2 production with optimized N/P ratio and PEI-mediated transfection results in low toxicity and high titer for in vitro and in vivo applications
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51. Variations in NPHP5 in Patients With Nonsyndromic Leber Congenital Amaurosis and Senior-Loken Syndrome
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52. Correlation between Photoreceptor Layer Integrity and Visual Function in Patients with Stargardt Disease: Implications for Gene Therapy
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55. Long-term Structural Outcomes of Late-stage RPE65 Gene Therapy
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56. Gene Therapy in Inherited Retinal Diseases: An Update on Current State of the Art
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57. The safety and efficacy of gene therapy treatment for monogenic retinal and optic nerve diseases: A systematic review
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59. Gene Therapeutic Approach Using Mutation-adapted U1 snRNA to Correct a RPGR Splice Defect in Patient-derived Cells
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60. Strategies to Modulate Immune Responses: A New Frontier for Gene Therapy
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61. Neuroprotective Strategies for the Treatment of Inherited Photoreceptor Degeneration
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68. Adeno-Associated Viral Gene Therapy for Inherited Retinal Disease
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69. Ocular Disease Therapeutics: Design and Delivery of Drugs for Diseases of the Eye
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70. RPE65, Visual Cycle Retinol Isomerase, Is Not Inherently 11-cis-specific
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71. RPE65 gene therapy slows cone loss in Rpe65-deficient dogs
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74. Clinical applications of gene therapy for rare diseases: A review
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75. Rationale and strategies for the development of safe and effective optimized AAV vectors for human gene therapy
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76. Gene Therapy Rescues Cone Structure and Function in the 3-Month-Old rd12 Mouse: A Model for Midcourse RPE65 Leber Congenital Amaurosis
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82. U1 snRNA-mediated gene therapeutic correction of splice defects caused by an exceptionally mild BBS mutation
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93. Human Retinal Disease from AIPL1 Gene Mutations: Foveal Cone Loss with Minimal Macular Photoreceptors and Rod Function Remaining
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