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

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11. Extracellular Vesicles Administered via Intrathecal Injection Mediate Safe Delivery of Nucleic Acids to the Central Nervous System for Gene Therapy
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12. Optogenetic recruitment of spinal reflex pathways from large‐diameter primary afferents in non‐transgenic rats transduced with AAV9/Channelrhodopsin 2
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13. Treatment of adult metachromatic leukodystrophy model mice using intrathecal administration of type 9 AAV vector encoding arylsulfatase A
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15. Assessment of AAV9 distribution and transduction in rats after administration through Intrastriatal, Intracisterna magna and Lumbar Intrathecal routes
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16. Intracerebroventricular delivery of self-complementary adeno-associated virus serotype 9 to the adult rat brain
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17. Advances in viral vector-based delivery systems for gene therapy: a comprehensive review
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18. Gene‐based therapies of neuromuscular disorders: an update and the pivotal role of patient organizations in their discovery and implementation
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20. A Comprehensive Map of CNS Transduction by Eight Recombinant Adeno-associated Virus Serotypes Upon Cerebrospinal Fluid Administration in Pigs
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21. Advanced Gene-Targeting Therapies for Motor Neuron Diseases and Muscular Dystrophies
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22. Gene Therapy: The View from NCATS
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23. Prevalence of Anti-Adeno-Associated Virus Serotype 9 Antibodies in Adult Patients with Spinal Muscular Atrophy
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24. Numerical study of intrathecal drug delivery to a permeable spinal cord: effect of catheter position and angle
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25. Breaking the sound barrier: Towards next-generation AAV vectors for gene therapy of hearing disorders
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26. Gene-targeted therapy for neurofibromatosis and schwannomatosis: The path to clinical trials
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27. Chondroitin Sulfate is the Primary Receptor for a Peptide-Modified AAV That Targets Brain Vascular Endothelium In Vivo
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28. Adeno-Associated Virus Delivery Limitations for Neurological Indications
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29. Pre-clinical delivery of gene therapy products to the cerebrospinal fluid: challenges and considerations for clinical translation
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30. In vivo selection in non-human primates identifies AAV capsids for on-target CSF delivery to spinal cord
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31. Neuronal genetic rescue normalizes brain network dynamics in a lysosomal storage disorder despite persistent storage accumulation
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32. Prevalence of Neutralizing Antibodies against Adeno-Associated Virus Serotypes 1, 2, and 9 in Non-Injected Latin American Patients with Heart Failure—ANVIAS Study
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33. Assessment of Safety and Biodistribution of AAVrh.10hCLN2 Following Intracisternal Administration in Nonhuman Primates for the Treatment of CLN2 Batten Disease
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34. Positron Emission Tomography Quantitative Assessment of Off-Target Whole-Body Biodistribution of I-124-Labeled Adeno-Associated Virus Capsids Administered to Cerebral Spinal Fluid
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35. High cerebrospinal fluid levels of interleukin-10 attained by AAV in dogs
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36. Reversibility and therapeutic development for neurodevelopmental disorders, insights from genetic animal models
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37. Mucopolysaccharidosis IIIB confers enhanced neonatal intracranial transduction by AAV8 but not by 5, 9 or rh10
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38. Current trends in gene therapy to treat inherited disorders of the brain
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39. Extension of the Lifespan of a Mouse Model of Rett Syndrome by Intracerebroventricular Delivery of MECP2
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40. Implantable and transdermal polymeric drug delivery technologies for the treatment of central nervous system disorders
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41. Investigating Immune Responses to the scAAV9-HEXM Gene Therapy Treatment in Tay–Sachs Disease and Sandhoff Disease Mouse Models
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42. Boron Delivery to Brain Cells via Cerebrospinal Fluid (CSF) Circulation for BNCT in a Rat Melanoma Model
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45. Emerging Concepts in Vector Development for Glial Gene Therapy: Implications for Leukodystrophies
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46. Pharmacokinetics of AAV9 Mediated Trastuzumab Expression in Rat Brain Following Systemic and Local Administration
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47. Impact of pre-existing immunity on safety and biodistribution of a single AAV9 vector intrathecal injection in cynomolgus monkeys
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48. Paving the way for future gene therapies: A case study of scientific spillover from delandistrogene moxeparvovec
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49. Adeno-associated virus vector hydrogel formulations for brain cancer gene therapy applications
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51. Initial gene vector dosing for studying symptomatology of amyotrophic lateral sclerosis in non‐human primates
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52. Intrathecal AAVrh10 corrects biochemical and histological hallmarks of mucopolysaccharidosis VII mice and improves behavior and survival
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53. Biodistribution of Adeno-Associated Virus Serotype 5 Viral Vectors Following Intrathecal Injection
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56. Intravenous immunoglobulin prevents peripheral liver transduction of intrathecally delivered AAV vectors
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57. Intrathecal delivery of a bicistronic AAV9 vector expressing β-hexosaminidase A corrects Sandhoff disease in a murine model: A dosage study
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61. Planet of the AAVs: The Spinal Cord Injury Episode
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62. Advances in the treatment of neuronal ceroid lipofuscinosis
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63. Motor Neuron Transduction After Intracisternal Delivery of AAV9 in a Cynomolgus Macaque
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64. Intracisternal delivery of NFκB-inducible scAAV2/9 reveals locoregional neuroinflammation induced by systemic kainic acid treatment
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66. Gene therapy for GM1 gangliosidosis: challenges of translational medicine.
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70. Clinical gene therapy development for the central nervous system: Candidates and challenges for AAVs
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84. Rescue of myocytes and locomotion through AAV2/9-2YF intracisternal gene therapy in a rat model of creatine transporter deficiency
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88. Comparison of different promoters to improve AAV vector-mediated gene therapy for neuronopathic Gaucher disease
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89. Riboflavin transporter deficiency: AAV9-SLC52A2 gene therapy as a new therapeutic strategy
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90. Viral Vector Technologies and Strategies: Improving on Nature
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93. Should patients with pre-existing anti-AAV antibodies be excluded from receiving intrathecally delivered AAV vectors?
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94. Cross-species efficacy of AAV-mediated ARSA replacement for metachromatic leukodystrophy
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95. Selective RNAi-silencing of Schwann cell Piezo1 alleviates mechanical hypersensitization following peripheral nerve injury
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97. Dynein-regulated brain transduction of AAV.CAP-B10 via cerebral lateral ventricle enhances hippocampal function after traumatic brain injury through Ngf gene delivery in mice
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