Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates

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3. Widespread transduction of astrocytes and neurons in the mouse central nervous system after systemic delivery of a self-complementary AAV-PHP.B vector
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6. Enhanced CNS transduction from AAV.PHP.eB infusion into the cisterna magna of older adult rats compared to AAV9
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9. Adeno-Associated Virus Neutralizing Antibodies in Large Animals and Their Impact on Brain Intraparenchymal Gene Transfer
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11. Adeno‐associated virus serotype 1‐based gene therapy for FTD caused by GRN mutations
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12. Gene-Editing Technologies Paired With Viral Vectors for Translational Research Into Neurodegenerative Diseases
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14. Biochemical, histological and functional correction of mucopolysaccharidosis Type IIIB by intra-cerebrospinal fluid gene therapy
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15. Recent advances in gene therapy for lysosomal storage disorders
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16. Restoration of Cytoskeleton Homeostasis After Gigaxonin Gene Transfer for Giant Axonal Neuropathy
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17. Disease correction by AAV-mediated gene therapy in a new mouse model of mucopolysaccharidosis type IIID
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18. Intrathecal administration of AAV/GALC vectors in 10-11-day-old twitcher mice improves survival and is enhanced by bone marrow transplant
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19. Efficacy and Safety of a Krabbe Disease Gene Therapy
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20. Enhanced Efficacy and Increased Long-Term Toxicity of CNS-Directed, AAV-Based Combination Therapy for Krabbe Disease
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21. Intrathecal sc-AAV9-CB-GFP: Systemic Distribution Predominates Following Single-Dose Administration in Cynomolgus Macaques
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22. Translating SOD1 Gene Silencing toward the Clinic: A Highly Efficacious, Off-Target-free, and Biomarker-Supported Strategy for fALS
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23. Designing and optimizing AAV-mediated gene therapy for neurodegenerative diseases: from bench to bedside
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24. Intrathecal AAV9/AP4M1 gene therapy for hereditary spastic paraplegia 50 shows safety and efficacy in preclinical studies
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25. Efficient central nervous system AAVrh10-mediated intrathecal gene transfer in adult and neonate rats
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26. Delivering AAV to the Central Nervous and Sensory Systems
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27. AAV9/MFSD8 gene therapy is effective in preclinical models of neuronal ceroid lipofuscinosis type 7 disease
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28. The Challenge of Gene Therapy for Neurological Diseases: Strategies and Tools to Achieve Efficient Delivery to the Central Nervous System
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29. Intrathecal Adeno-Associated Viral Vector-Mediated Gene Delivery for Adrenomyeloneuropathy
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30. Translational Approaches for Brain Delivery of Biologics via Cerebrospinal Fluid
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31. Advances in the treatment of neuronal ceroid lipofuscinosis
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32. Delivery of an Adeno-Associated Virus Vector into Cerebrospinal Fluid Attenuates Central Nervous System Disease in Mucopolysaccharidosis Type II Mice
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33. Human in silico trials for parametric computational fluid dynamics investigation of cerebrospinal fluid drug delivery: impact of injection location, injection protocol, and physiology
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34. Engineering viral vectors for acoustically targeted gene delivery
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35. Novel Vector Design and Hexosaminidase Variant Enabling Self-Complementary Adeno-Associated Virus for the Treatment of Tay-Sachs Disease
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36. Dual-isoform hUBE3A gene transfer improves behavioral and seizure outcomes in Angelman syndrome model mice
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37. Translation of MicroRNA-Based Huntingtin-Lowering Therapies from Preclinical Studies to the Clinic.
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38. Overcoming delivery barriers in immunotherapy for glioblastoma
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39. Preclinical biodistribution, tropism, and efficacy of oligotropic AAV/Olig001 in a mouse model of congenital white matter disease
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40. Physical positioning markedly enhances brain transduction after intrathecal AAV9 infusion
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41. Quantitative Whole-Body Imaging of I-124-Labeled Adeno-Associated Viral Vector Biodistribution in Nonhuman Primates
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42. Intracisternal delivery of AAV9 results in oligodendrocyte and motor neuron transduction in the whole central nervous system of cats
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43. Enhancing gene transfer to renal tubules and podocytes by context-dependent selection of AAV capsids
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44. Gene therapy approaches targeting Schwann cells for demyelinating neuropathies
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46. Intracerebroventricular Administration of AAV9-PHP.B SYN1-EmGFP Induces Widespread Transgene Expression in the Mouse and Monkey Central Nervous System
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47. Implications of mRNA translation dysregulation for neurological disorders
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48. Blood-brain barrier delivery for lysosomal storage disorders with IgG-lysosomal enzyme fusion proteins
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49. Systemic Gene Transfer of a Hexosaminidase Variant Using an scAAV9.47 Vector Corrects G M2 Gangliosidosis in Sandhoff Mice
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50. Seven-year follow-up of durability and safety of AAV CNS gene therapy for a lysosomal storage disorder in a large animal
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51. Recombinant Adeno-Associated Virus Vectors for Gene Therapy of the Central Nervous System: Delivery Routes and Clinical Aspects
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52. Intrathecal administration of IGF-I by AAVrh10 improves sensory and motor deficits in a mouse model of diabetic neuropathy
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53. High throughput screening of novel AAV capsids identifies variants for transduction of adult NSCs within the subventricular zone
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54. Adeno-associated viral vector serotype 9-based gene replacement therapy for SURF1-related Leigh syndrome
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55. Efficacy of AAV serotypes to target Schwann cells after intrathecal and intravenous delivery
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56. Adeno-associated virus serotypes 9 and rh10 mediate strong neuronal transduction of the dog brain
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57. Viral vectors for gene delivery to the inner ear
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58. The AAV9 Variant Capsid AAV-F Mediates Widespread Transgene Expression in Nonhuman Primate Spinal Cord After Intrathecal Administration
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59. A Single Injection of Recombinant Adeno-Associated Virus into the Lumbar Cistern Delivers Transgene Expression Throughout the Whole Spinal Cord
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60. Pre-clinical Gene Therapy with AAV9/AGA in Aspartylglucosaminuria Mice Provides Evidence for Clinical Translation
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61. Intrathecal delivery and its applications in leptomeningeal disease
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62. Mucopolysaccharidosis-like phenotype in feline Sandhoff disease and partial correction after AAV gene therapy
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63. Efficacy of a Bicistronic Vector for Correction of Sandhoff Disease in a Mouse Model
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64. In Vivo Gene Therapy for Mucopolysaccharidosis Type III (Sanfilippo Syndrome): A New Treatment Horizon
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65. Adeno-Associated Viral Vectors as Versatile Tools for Parkinson’s Research, Both for Disease Modeling Purposes and for Therapeutic Uses
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66. Using Patient-Specific Induced Pluripotent Stem Cells and Wild-Type Mice to Develop a Gene Augmentation-Based Strategy to Treat CLN3 -Associated Retinal Degeneration
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67. A numerical investigation of intrathecal isobaric drug dispersion within the cervical subarachnoid space
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68. Nanocarriers for Delivery of Oligonucleotides to the CNS
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69. Immune responses to central nervous system directed adeno-associated virus gene therapy: Does direct CNS delivery make a difference?
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70. Trends and challenges of AAV-delivered gene editing therapeutics for CNS disorders: Implications for neurodegenerative disease
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71. Recent Advances in RNA Interference Therapeutics for CNS Diseases
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73. Atomic structure of a rationally engineered gene delivery vector, AAV2. 5
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75. Genetic and Epigenetic Landscape for Drug Development in Polycystic Ovary Syndrome
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76. A Single Injection of an Optimized Adeno-Associated Viral Vector into Cerebrospinal Fluid Corrects Neurological Disease in a Murine Model of GM1 Gangliosidosis
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83. Gene therapy for the neurological manifestations in lysosomal storage disorders
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85. Adeno-associated virus-mediated delivery of CRISPR-Cas9 for genome editing in the central nervous system
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86. Improved systemic AAV gene therapy with a neurotrophic capsid in Niemann–Pick disease type C1 mice
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87. Emerging Perspectives on Gene Therapy Delivery for Neurodegenerative and Neuromuscular Disorders
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88. Charcot–Marie–Tooth neuropathies: Current gene therapy advances and the route toward translation
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90. Cellular unfolded protein response against viruses used in gene therapy
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91. Gold nanocarriers for transport of oligonucleotides across brain endothelial cells
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92. Characterization of intrathecal cerebrospinal fluid geometry and dynamics in cynomolgus monkeys (macaca fascicularis) by magnetic resonance imaging
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93. Preclinical Toxicity Evaluation of AAV for Pain: Evidence from Human AAV Studies and from the Pharmacology of Analgesic Drugs
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94. Advances in AAV-mediated gene replacement therapy for pediatric monogenic neurological disorders
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95. Applications of adeno-associated virus vector-mediated gene delivery for neurodegenerative diseases and psychiatric diseases: Progress, advances, and challenges
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96. Delivery of therapeutic AAV9 vectors via cisterna magna to treat neurological disorders
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97. Pilot Study Assessing the Impact of Intrathecal Administration of Variants AAV-PHP.B and AAV-PHP.eB on Brain Transduction in Adult Rhesus Macaques
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98. Comparison of Endovascular and Intraventricular Gene Therapy With Adeno-Associated Virus–α-L-Iduronidase for Hurler Disease
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99. Normalization and Improvement of CNS Deficits in Mice With Hurler Syndrome After Long-term Peripheral Delivery of BBB-targeted Iduronidase
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100. Access to cerebrospinal fluid in piglets via the cisterna magna: optimization and description of the technique
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