Global CNS Transduction of Adult Mice by Intravenously Delivered rAAVrh.8 and rAAVrh.10 and Nonhuman Primates by rAAVrh.10

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Items 101–200 of 213

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2. Fighting against kidney diseases with small interfering RNA: opportunities and challenges
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3. Emerging therapies for neuropathic lysosomal storage disorders
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4. Adeno-Associated Viral Vectors as Versatile Tools for Parkinson’s Research, Both for Disease Modeling Purposes and for Therapeutic Uses
   Fajardo-Serrano, Ana, Rico, Alberto J., Roda, Elvira, Honrubia, Adriana, Arrieta, Sandra, Ariznabarreta, Goiaz, Chocarro, Julia, Lorenzo-Ramos, Elena, Pejenaute, Alvaro, Vázquez, Alfonso et al. · 2021 · International Journal of Molecular Sciences
5. Drug Delivery Strategies to Overcome the Blood–Brain Barrier (BBB)
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6. Nucleic acid therapies for CNS diseases: Pathophysiology, targets, barriers, and delivery strategies
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7. An Engineered Galactosylceramidase Construct Improves AAV Gene Therapy for Krabbe Disease in Twitcher Mice
   Xiufang Pan, Scott A. Sands, Yongping Yue, Keqing Zhang, Steven M. LeVine, Dongsheng Duan · 2019 · Human Gene Therapy
8. Novel AAV44.9-Based Vectors Display Exceptional Characteristics for Retinal Gene Therapy
   Boye, Sanford L., Choudhury, Shreyasi, Crosson, Sean, Di Pasquale, Giovanni, Afione, Sandra, Mellen, Russell, Makal, Victoria, Calabro, Kaitlyn R., Fajardo, Diego, Peterson, James et al. · 2020 · Molecular Therapy
9. Global CNS correction in a large brain model of human alpha-mannosidosis by intravascular gene therapy
   Sea Young Yoon, Jacqueline E. Hunter, Sanjeev Chawla, Dana L. Clarke, Caitlyn M. Molony, Patricia A. O’Donnell, Jessica H. Bagel, Manoj Kumar, Harish Poptani, Charles H. Vite et al. · 2020 · Brain
10. Innovative Treatments for Lysosomal Diseases
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11. Rapid generation of mouse model for emerging infectious disease with the case of severe COVID-19
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12. Gene Therapy for the Treatment of Neurological Disorders: Metabolic Disorders
    Dominic J. Gessler, Guangping Gao · 2015 · Methods in molecular biology
13. Scientific and Regulatory Policy Committee Points to Consider: Nonclinical Research and Development of In Vivo Gene Therapy Products, Emphasizing Adeno-Associated Virus Vectors
    Julie A. Hutt, Basel T. Assaf, Brad N. Bolon, Joy A. Cavagnaro, Elizabeth J. Galbreath, Branka Grubor, Lisa M. Kattenhorn, Annette Romeike, Laurence O. Whiteley · 2021 · Toxicologic Pathology
14. Functional Analysis of the Putative Integrin Recognition Motif on Adeno-associated Virus 9
    Shen Shen, Garrett E. Berry, Ruth M. Castellanos Rivera, Roland Cheung, Andrew N. Troupes, Sarah M. Brown, Tal Kafri, Aravind Asokan · 2014 · Journal of Biological Chemistry
15. Leukodystrophies
    Marjo S. van der Knaap, Nicole I. Wolf, Vivi Majella Heine · 2016 · Neurology Clinical Practice
16. Adeno-associated virus serotype rh.10 displays strong muscle tropism following intraperitoneal delivery
    Jianzhong Ai, Jia Li, Dominic J. Gessler, Qin Su, Qiang Wei, Hong Li, Guangping Gao · 2017 · Scientific Reports
17. Slow Intrathecal Injection of rAAVrh10 Enhances its Transduction of Spinal Cord and Therapeutic Efficacy in a Mutant SOD1 Model of ALS
    Dongxiao Li, Chong Liu, Chunxing Yang, Dan Wang, Dongxia Wu, Yinkuang Qi, Qin Su, Guangping Gao, Zuoshang Xu, Yansu Guo · 2017 · Neuroscience
18. Systemic AAV6-synapsin-GFP administration results in lower liver biodistribution, compared to AAV1&2 and AAV9, with neuronal expression following ultrasound-mediated brain delivery
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19. Clade F AAVHSCs cross the blood brain barrier and transduce the central nervous system in addition to peripheral tissues following intravenous administration in nonhuman primates
    Jeff L. Ellsworth, Jacinthe Gingras, Laura J. Smith, Hillard L. Rubin, Tania A. Seabrook, Kruti Patel, Nicole Zapata, Kevin C. Olivieri, Michael O’Callaghan, Elizabeth A. Chlipala et al. · 2019 · PLoS ONE
20. rAAV Gene Therapy in a Canavan’s Disease Mouse Model Reveals Immune Impairments and an Extended Pathology Beyond the Central Nervous System
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21. RNA therapies for CNS diseases
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22. Neurosurgical gene therapy for central nervous system diseases
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23. Gene therapy in glioblastoma multiforme: Can it be a role changer?
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24. Adeno Associated Viral Vector Delivered RNAi for Gene Therapy of SOD1 Amyotrophic Lateral Sclerosis
    Lorelei Stoica, Miguel Sena‐Esteves · 2016 · Frontiers in Molecular Neuroscience
25. Efficient gene delivery to photoreceptors using AAV2/rh10 and rescue of the Rho−/− mouse
    Arpad Palfi, Naomi Chadderton, Mary O’Reilly, Kerstin Nagel‐Wolfrum, Uwe Wolfrum B, Jean Bennett, Peter Humphries, Paul F. Kenna, Sophia Millington‐Ward, Jane Farrar · 2015 · Molecular Therapy — Methods & Clinical Development
26. Neurological glycogen storage diseases and emerging therapeutics
    Matthieu G. Colpaert, Pankaj Kumar Singh, Katherine J. Donohue, Natacha T Pires, David Dwight Fuller, Manuela Corti, Barry J. Byrne, Ramon C. Sun, Craig W. Vander Kooi, Matthew S. Gentry · 2024 · Neurotherapeutics
27. Aspartylglucosaminuria: Clinical Presentation and Potential Therapies
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28. Charcot–Marie–Tooth neuropathies: Current gene therapy advances and the route toward translation
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29. Optimization of Dexamethasone Administration for Maintaining Global Transduction Efficacy of Adeno-Associated Virus Serotype 9
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30. Engineered viral vectors for functional interrogation, deconvolution, and manipulation of neural circuits
    Sabrina Sun, David V. Schaffer · 2018 · Current Opinion in Neurobiology
31. Perinatal systemic gene delivery using adeno-associated viral vectors
    Rajvinder Karda, Suzanne M. K. Buckley, Citra Nurfarah Zaini Mattar, Joanne Ng, Giulia Massaro, Michael P. Hughes, Manju A. Kurian, Julien Baruteau, Paul Gissen, Jerry Kok Yen Chan et al. · 2014 · Frontiers in Molecular Neuroscience
32. Advances in AAV-mediated gene replacement therapy for pediatric monogenic neurological disorders
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33. 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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34. Attenuation of the Niemann-Pick type C2 disease phenotype by intracisternal administration of an AAVrh.10 vector expressing Npc2
    Sandra Markmann, Jasmine J. Christie-Reid, Jonathan B. Rosenberg, Bishnu P. De, Stephen M. Kaminsky, Ronald G. Crystal, Dolan Sondhi · 2018 · Experimental Neurology
35. Intravenous delivery of adeno-associated viral gene therapy in feline GM1 gangliosidosis
    Amanda L. Gross, Heather L. Gray‐Edwards, Cassie N Bebout, Nathan L. Ta, Kayly Nielsen, Brandon L. Brunson, Kalajan R Lopez Mercado, Devin E. Osterhoudt, Ana Rita Batista, Stacy A. Maitland et al. · 2021 · Brain
36. Canavan Disease as a Model for Gene Therapy-Mediated Myelin Repair
    Anoushka Lotun, Dominic J. Gessler, Guangping Gao · 2021 · Frontiers in Cellular Neuroscience
37. Understanding the Potential of Genome Editing in Parkinson’s Disease
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38. A novel strategy for delivering N iemann‐ P ick type C2 proteins across the blood–brain barrier using the brain endothelial‐specific AAV‐BR1 virus
    Charlotte Laurfelt Munch Rasmussen, Eva Hede, Lisa Greve Routhe, Jakob Körbelin, Steinunn Sara Helgudóttir, Louiza Bohn Thomsen, Markus Schwaninger, Annette Burkhart, Torben Moos · 2022 · Journal of Neurochemistry
39. Gene therapy of the central nervous system: General considerations on viral vectors for gene transfer into the brain
    Ché Serguera, Alexis‐Pierre Bemelmans · 2014 · Revue Neurologique
40. Intravenous delivery for treatment of mucopolysaccharidosis type I: A comparison of AAV serotypes 9 and rh10
    Lalitha R. Belur, Kelly M. Podetz-Pedersen, Thuy An Tran, Joshua A. Mesick, Nathaniel M. Singh, Maureen S. Riedl, Lucy Vulchanova, Karen Kozarsky, R. Scott McIvor · 2020 · Molecular Genetics and Metabolism Reports
41. Survival benefit and phenotypic improvement by hamartin gene therapy in a tuberous sclerosis mouse brain model
    Shilpa Prabhakar, Xuan Zhang, June Goto, Sangyeul Han, Charles Pin‐Kuang Lai, Roderick Terry Bronson, Miguel Sena‐Esteves, Vijaya Ramesh, Anat O Stemmer-Rachamimov, David J. Kwiatkowski et al. · 2015 · Neurobiology of Disease
42. Genetic manipulation of brain endothelial cells in vivo
    Julian Christopher Assmann, Jakob Körbelin, Markus Schwaninger · 2015 · Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease
43. Emerging trends in virus and virus-like particle gene therapy delivery to the brain
    Heshadi Primrose Primrose Mandalawatta, Rajendra KC, Kirsten A. Fairfax, Alex William Hewitt · 2024 · Molecular Therapy — Nucleic Acids
44. Unique Glycan Signatures Regulate Adeno-Associated Virus Tropism in the Developing Brain
    Giridhar Murlidharan, Travis Corriher, H. Troy Ghashghaei, Aravind Asokan · 2015 · Journal of Virology
45. A Comprehensive Map of CNS Transduction by Eight Recombinant Adeno-associated Virus Serotypes Upon Cerebrospinal Fluid Administration in Pigs
    Nicolina Cristina Sorrentino, Veronica Maffia, Sandra Strollo, Vincenzo Cacace, Noemi Romagnoli, Anna Manfredi, Domenico Ventrella, Francesco Dondi, Francesca Barone, Massimo Giunti et al. · 2015 · Molecular Therapy
46. Potential of microRNAs as novel targets in the alleviation of pathological fear
    Conor P. Murphy, Nicolas Singewald · 2017 · Genes Brain & Behavior
47. Capsid-Engineering for Central Nervous System-Directed Gene Therapy with Adeno-Associated Virus Vectors
    Josephine Macdonald, Jennifer Marx, Hildegard Büning · 2021 · Human Gene Therapy
48. Direct Intrathecal Injection of Recombinant Adeno-associated Viruses in Adult Mice
    Dongxiao Li, Yundu Li, Yunyun Tian, Zuoshang Xu, Yansu Guo · 2019 · Journal of Visualized Experiments
49. Distributional comparison of different AAV vectors after unilateral cochlear administration
    Shuang Han, Zhijiao Xu, Shengyi Wang, Honghai Tang, Shaowei Hu, Hui Wang, Guofang Guan, Yilai Shu · 2023 · Gene Therapy
50. Improved gene delivery to adult mouse spinal cord through the use of engineered hybrid adeno-associated viral serotypes
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51. Efficiency of cell-type specific and generic promoters in transducing oxytocin neurons and monitoring their neural activity during lactation
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52. Systemic gene therapy for methylmalonic acidemia using the novel adeno-associated viral vector 44.9
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53. On the development of optical peripheral nerve interfaces
    Anderson, HansE., Weir, RichardF. ff. · 2019 · Neural Regeneration Research
54. Identification of peripheral oxytocin‐expressing cells using systemically applied cell‐type specific adeno‐associated viral vector
    Luis Paiva, Maja Lozić, Andrew J. Allchorne, Valery Grinevich, Mike Ludwig · 2021 · Journal of Neuroendocrinology
55. Progress in gene and cell therapies for the neuronal ceroid lipofuscinoses
    Anthony Donsante, Nicholas M. Boulis · 2018 · Expert Opinion on Biological Therapy
56. Therapeutic potential of combined viral transduction and CRISPR/Cas9 gene editing in treating neurodegenerative diseases
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57. Structure of the AAVhu.37 capsid by cryoelectron microscopy
    Jason T. Kaelber, Samantha A. Yost, Keith Webber, Emre Firlar, Ye Liu, Olivier Danos, Andrew C. Mercer · 2020 · Acta Crystallographica Section F Structural Biology Communications
58. Chondroitin Sulfate is the Primary Receptor for a Peptide-Modified AAV That Targets Brain Vascular Endothelium In Vivo
    James C. Geoghegan, Nicholas W. Keiser, Anna Okulist, Inês Martins, Matthew S Wilson, Beverly L. Davidson · 2014 · Molecular Therapy — Nucleic Acids
59. An engineered adeno-associated virus mediates efficient blood-brain barrier penetration with enhanced neurotropism and reduced hepatotropism
    Nengsong Luo, Kunzhang Lin, Yuxiang Cai, Zilian Zhang, Xiaokai Sui, Zengpeng Han, Wenjia Yuan, Jiayong Xing, Zhu Gui, Gangning Liu et al. · 2025 · Journal of Controlled Release
60. Noncoding RNA therapeutics for substance use disorder
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61. Gene and Cellular Therapies for Leukodystrophies
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62. Mucopolysaccharidosis IIIB confers enhanced neonatal intracranial transduction by AAV8 but not by 5, 9 or rh10
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63. A brief guide for gene delivery to the brain using adeno-associated viral vectors
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64. Emerging Concepts in Vector Development for Glial Gene Therapy: Implications for Leukodystrophies
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65. A Review of Brain-Targeted Nonviral Gene-Based Therapies for the Treatment of Alzheimer’s Disease
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66. Adeno-associated virus vector hydrogel formulations for brain cancer gene therapy applications
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67. Brain endothelial cell‐targeted gene therapy of neurovascular disorders
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68. Stem Cell Applications in Lysosomal Storage Disorders: Progress and Ongoing Challenges
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69. Single Systemic Administration of a Gene Therapy Leading to Disease Treatment in Metachromatic LeukodystrophyArsaKnock-Out Mice
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70. The G to A transformation of rs4702 polymorphism in 3’UTR of FURIN reduced the risk of radiotherapy‐induced cognitive impairment in glioma patients
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71. Intravenous AAV9- ATP7A plus subcutaneous copper histidinate optimizes outcomes in a lethal Menkes disease mouse model
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72. Therapeutic Strategy for Fabry Disease by Intravenous Administration of Adeno-Associated Virus 9 in a Symptomatic Mouse Model
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73. Making the White Matter Matters: Progress in Understanding Canavan’s Disease and Therapeutic Interventions Through Eight Decades
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74. Gene Therapy for Rett Syndrome: Prospects and Challenges
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75. Protein modeling and clinical description of a novel in‐frame GLB 1 deletion causing GM 1 gangliosidosis type II
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76. Generation of light-producing somatic-transgenic mice using adeno-associated virus vectors
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77. Present insights into the progress in gene therapy delivery systems for central nervous system diseases
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78. Stem cell and gene therapies for leukodystrophies
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79. Viral vectors for gene delivery to the central nervous system
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80. New Insights: MicroRNA Function in CNS Development and Psychiatric Diseases
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81. New insights into tuberous sclerosis complex: from structure to pathogenesis
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82. Vector Tropism
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83. An automated method for the quantification of transgene expression in motor axons of the peripheral nerve
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84. Intravascular injections of adenoassociated viral vector serotypes rh10 and PHP.B transduce murine sciatic nerve axons
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85. Progress and challenges in intrathecal gene therapy for neurological disorders
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86. Adeno-associated Virus-Mediated Gene Delivery Across the Blood-Brain Barrier
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87. Oligodendrocytes, the Forgotten Target of Gene Therapy
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88. Crossing the Blood-Brain Barrier with AAVs: What’s After SMA?
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89. Engineered AAVs for Non-Invasive Functional Transgene Expression in Rodent and Non-Human Primate Central and Peripheral Nervous Systems
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90. Applications and Developments of Gene Therapy Drug Delivery Systems for Neurological Disorders
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92. Viral Vector Gene Delivery to the Brain for Treating Neurogenetic Diseases
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94. Generation of light-producing somatic-transgenic mice using adeno-associated virus vectors
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96. Viral vectors for gene therapy
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98. Viral Vector Therapeutics Against Alzheimer’s Disease
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99. Biology of AAV Vectors in the Central Nervous System
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100. Virus-mediated Delivery of the Fmr1 Gene as a Tool for the Treatment of Fragile X Syndrome
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