Several rAAV Vectors Efficiently Cross the Blood–brain Barrier and Transduce Neurons and Astrocytes in the Neonatal Mouse Central Nervous System

Zhang, Hongwei, Yang, Bin, Mu, Xin, Ahmed, Seemin Seher, Su, Qin, He, Ran, Wang, Hongyan, Mueller, Christian +4 more·2011·Molecular Therapy

Cited by (291)

Items 201–291 of 291

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  2. Optimized AAV capsids for basal ganglia diseases show robust potency and distribution
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  3. A new protocol for whole-brain biodistribution analysis of AAVs by tissue clearing, light-sheet microscopy and semi-automated spatial quantification
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  5. Selective Neuronal Uptake and Distribution of AAVrh8, AAV9, and AAVrh10 in Sheep After Intra-Striatal Administration
    Erica Mondo, Richard P. Moser, Guangping Gao, Christian Mueller, Miguel Sena‐Esteves, Ellen Sapp, Edith L. Pfister, D. C. O'Connell, Kendra A. Takle, Kirsten E. Erger et al. · 2018 · Journal of Huntington s Disease
  6. Intrathecal AAVrh10 corrects biochemical and histological hallmarks of mucopolysaccharidosis VII mice and improves behavior and survival
    Gemma Pagès, Lydia Giménez‐Llort, Belén García-Lareu, Lorena Ariza, Marc Navarro, Caty Casas, Miguel Chillón, Assumpció Bosch · 2019 · Human Molecular Genetics
  7. Viral Vector Delivery of DREADDs for CNS Therapy
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  8. Slow Infusion of Recombinant Adeno-Associated Viruses into the Mouse Cerebrospinal Fluid Space
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  9. Tissue blood volumes and weights in adult beagle dogs.
    R.G. Cuddihy, Russell P. Hall, CHARLES H. HOBBS, Bruce B. Boecker, Bruce A. Muggenburg · 1972 · OSTI OAI (U.S. Department of Energy Office of Scientific and Technical Information)
  10. The potential of gene delivery for the treatment of traumatic brain injury
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  11. Challenges for Gene Therapy of CNS Disorders and Implications for Parkinson's Disease Therapies
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  12. Cryoprecipitate augments the global transduction of the adeno-associated virus serotype 9 after a systemic administration
    Zheng Chai, Xintao Zhang, Kelly Michelle Rigsbee, Mei Wang, Richard Jude Samulski, Chengwen Li · 2018 · Journal of Controlled Release
  13. In utero delivery of rAAV2/9 induces neuronal expression of the transgene in the brain: towards new models of Parkinson’s disease
    Lucie Chansel‐Debordeaux, Mathieu Bourdenx, Sandra Doveró, Virginie Grouthier, Nathalie Dutheil, Agnès Espana, Laurent Groc, Clément Jimenez, Erwan Bézard, Benjamin Dehay · 2017 · Gene Therapy
  14. Considerations for the use of virally delivered genetic tools for in-vivo circuit analysis and behavior in mutant mice: a practical guide to optogenetics
    Dóra Zelena, Kornél Demeter, József Haller, Diána Balázsfi · 2017 · Behavioural Pharmacology
  15. Engineering brain-penetrant PROTACs: Bridging molecular design and CNS delivery
    Xinuo Li, Jinran Li, Yuan Sun, Tingting Yao, Yi Lu, Sai Liu, Haotian Li, Yiting Shi, Long Chen, Yongjun Zhao et al. · 2026 · Advanced Drug Delivery Reviews
  16. Serotype-dependent recombinant adeno-associated vector (AAV) infection of Epstein–Barr virus-positive B-cells, towards recombinant AAV-based therapy of focal EBV + lymphoproliferative disorders
    Elham Ahmadi, Mehrdad Ravanshad, Jun Xie, Rajesh Panigrahi, Sandeep Singh Jubbal, Santosh Kumar Guru, Gao Guangping, Mazyar Ziyaeyan, Joyce D. Fingeroth · 2021 · Virology Journal
  17. Clinical gene therapy development for the central nervous system: Candidates and challenges for AAVs
    Tiffany W. Leong, Arindam Pal, Qi Cai, Zhenghong Gao, Xiaoqing Li, Leonidas G. Bleris, Heather N. Hayenga, Zhenpeng Qin · 2023 · Journal of Controlled Release
  18. Therapeutic strategy for Fabry disease by intravenous administration of adeno‐associated virus 2 or 9 in α‐galactosidase A‐deficient mice
    Yuka Hayashi, Yoshihide Sehara, Ryota Watano, Kenji Ohba, Yuki Takayanagi, Kazuhiro Muramatsu, Yoshio Sakiyama, Hiroaki Mizukami · 2023 · The Journal of Gene Medicine
  19. Making the White Matter Matters: Progress in Understanding Canavan’s Disease and Therapeutic Interventions Through Eight Decades
    Seemin Seher Ahmed, Guangping Gao · 2014 · JIMD Reports
  20. Effects of gene therapy on cardiovascular symptoms of lysosomal storage diseases
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    Osita W. Ogujiofor, Iliodora V. Pop, Felipe Espinosa, Razaq O. Durodoye, Michael L. Viacheslavov, Rachel Jarvis, Mark A. Landy, Gord Fishell, Robert P. Machold, Helen C. Lai · 2021 · eNeuro
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    Kamal K.E. Gadalla, Paul D. Ross, Ralph D. Hector, Noha Gamal Bahey, Mark E.S. Bailey, Stuart Cobb · 2015 · Future Neurology
  23. AAV Gene Therapy Strategies for Lysosomal Storage Disorders with Central Nervous System Involvement
    Golebiowski, Diane, Bradbury, Allison M., Kwon, Churl-Su, van der Bom, Imramsjah M. J., Stoica, Lorelei, Johnson, Aime K., Wilson, Diane U., Gray-Edwards, Heather L., Hudson, Judith A., Johnson, Jacob A. et al. · 2015 · Neuromethods
  24. Protein Carrier Adeno-Associated Virus
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    Darren A. Murrey, Bartholomew J. Naughton, F. Jason Duncan, Aaron S. Meadows, Tierra Ware, Katie Campbell, William G. Bremer, Christopher M. Walker, Laurie Goodchild, Brad N. Bolon et al. · 2014 · Human Gene Therapy Clinical Development
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    Amel Karaa, Thomas Klopstock · 2023 · Handbook of clinical neurology
  27. Gene Therapy for Canavan's Disease Takes a Step Forward
    Seemin Seher Ahmed, Guangping Gao · 2013 · Molecular Therapy
  28. Present insights into the progress in gene therapy delivery systems for central nervous system diseases
    Zefeng Yang, Xinchi Jiang, Jianqing Gao · 2024 · International Journal of Pharmaceutics
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  31. Gene Therapy Methods and Their Applications in Neurological Disorders
    Mingjie Li, B. Joy Snider · 2018 · Elsevier eBooks
  32. Optimized AAV capsids for diseases of the basal ganglia show robust potency and distribution in adult nonhuman primates
    David E. Leib, YH Chen, Luis Tecedor, Paul T. Ranum, Megan S. Keiser, BC Lewandowski, Ellie M. Carrell, S Arora, Icnelia Huerta-Ocampo, Xing Liu et al. · 2024 · bioRxiv (Cold Spring Harbor Laboratory)
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  36. Transduction of Brain Neurons in Juvenile Chum Salmon (Oncorhynchus keta) with Recombinant Adeno-Associated Hippocampal Virus Injected into the Cerebellum during Long-Term Monitoring
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  38. Intravascular injections of adenoassociated viral vector serotypes rh10 and PHP.B transduce murine sciatic nerve axons
    Hans E. Anderson, K Schaller, John H. Caldwell, Richard F ff Weir · 2019 · Neuroscience Letters
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    Jayanth S. Chandran, Joseph M. Scarrott, Pamela J. Shaw, Mimoun Azzouz · 2017 · Advances in experimental medicine and biology
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    Yujia A. Chan, Benjamin E. Deverman · 2022 · AAPS advances in the pharmaceutical sciences series
  45. Mosaic AAV mediates efficient transduction across the central and peripheral nervous systems
    Min Jiang, Wenqing Yin, Xiaolu Mo, Zhounan Wang, Mengsheng S. Qiu, Zhong‐Min Dai · 2025 · Molecular Brain
  46. From Treatment to Cure
    Ivan Pavlov, Stéphanie Schorge · 2014 · International review of neurobiology
  47. Viral Vectors in Primate Research: Examples from Parkinson’s Disease Research
    Grégory Porras, Benjamin Dehay, Erwan Bézard · 2013 · Neuromethods
  48. Induced Cell Turnover: A Novel Therapeutic Modality for In Situ Tissue Regeneration
    Francesco Albert Bosco Cortese, Sebastian P. Aguiar, Giovanni Santostasi · 2017 · Human Gene Therapy
  49. Viral Vector Gene Delivery to the Brain for Treating Neurogenetic Diseases
    Linnet Ramos, Jacqueline E. Hunter, John H N Wolfe · 2017 · Springer eBooks
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    Victor Banerjee, Ofek Oren, Bar Dagan, Ran Taube, Stanislav Engel, Niv Papo · 2018 · ACS Chemical Neuroscience
  51. Modulating the Blood-Brain Barrier by Light Stimulation of Molecular-Targeted Nanoparticles
    Xiaoqing Li, Vamsidhara Vemireddy, Qi Cai, Hejian Xiong, Peiyuan Kang, Xiuying Li, Monica Giannotta, Heather N. Hayenga, Edward Pan, Shashank Ramesh Sirsi et al. · 2020 · bioRxiv (Cold Spring Harbor Laboratory)
  52. Gene therapy for Krabbe disease: evidence from mouse and canine models
    Yang Li, Xiang Dong, Jian Cheng Guo, Yafeng Lv · 2025 · Gene
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    Fushuang Zheng, Rongli Guan, Xiaojin Yu, Jiaxin Yang, Hai Zhao, Fan Yang · 2026 · Cellular & Molecular Biology Letters
  54. CNS Gene Therapy Utilizing Intravenously Administered Viral Vectors
    Adam K. Bevan, Brian K. Kaspar · 2013 · AAPS advances in the pharmaceutical sciences series
  55. Bioluminescence-Based Monitoring of Virus Vector-Mediated Gene Transfer in Mice
    Casey A. Maguire · 2013 · Methods in molecular biology
  56. Gene Therapy of CNS Disorders Using Recombinant AAV Vectors
    Giridhar Murlidharan, Richard Jude Samulski, Aravind Asokan · 2016 · Springer eBooks
  57. Gene therapy approaches in central nervous system regenerative medicine
    Assumpció Bosch, Miguel Chillón · 2020 · Elsevier eBooks
  58. Huntingtin lowering therapeutics
    Neil Aronin M. D, Miguel Sena‐Esteves, Anastasia Khvorova, Marian DiFiglia, Michael Brodsky · 2024 · Elsevier eBooks
  59. Next generation AAV-F capsid gene therapy rescues disease pathology in a model of pyruvate dehydrogenase complex deficiency
    Anna Keegan, Özge Çetin, Ellie M. Chilcott, Juan F. Antinao Diaz, Simon D. Eaton, Simon N. Waddington, John Robert Counsell, Shamima Rahman, Rajvinder Karda · 2026 · Molecular Therapy Advances
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    Tielin Chen, Honglian Wang, Yun-Hong Liu, Yin Fang, Ruizhi Tan, Puhui Zhou, Qin Zhou, Xiaoyan Lv · 2014 · The Scientific World JOURNAL
  61. Systemic Gene Therapy for Targeting the CNS
    Sara E. Gombash, Kevin D. Foust · 2015 · Methods in molecular biology
  62. Gene Therapy for Neurodevelopmental Disorders
    Douglas M. McCarty, Haiyan Fu · 2014 · Encyclopedia of Life Sciences
  63. Development of Therapies for Spinal Muscular Atrophy Using Gene Therapy and Nanotechnology
    Daniel Little · 2013 · White Rose eTheses Online (University of Leeds, The University of Sheffield, University of York)
  64. MPS IIIA: gene therapy for brain and body
    Michael John Haas · 2013 · Science-Business eXchange
  65. Gene Therapy for Stroke
    Fanxia Shen, Hua Su · 2017 · Translational medicine research
  66. Gene Transfer in Spinal Muscular Atrophy
    Kevin D. Foust, Brian K. Kaspar · 2016 · Elsevier eBooks
  67. Biology of AAV Vectors in the Central Nervous System
    Giridhar Murlidharan · 2019 · Carolina Digital Repository (University of North Carolina at Chapel Hill)
  68. Virus-mediated Delivery of the Fmr1 Gene as a Tool for the Treatment of Fragile X Syndrome
    Shervin Gholizadeh Moghaddam · 2016 · TSpace (University of Toronto)
  69. Intravenously administered gene therapy for neuronopathic Gaucher disease
    Giulia Massaro · 2018 · UCL Discovery (University College London)
  70. Adeno-Associated Virus Vector Mediated Gene Delivery to Neurons and Glial Cells in Damaged Spinal Cord
    Victor L. Arvanian, Hayk A. Petrosyan · 2018 · Elsevier eBooks
  71. Intravenous injection of AAV-PHP.eB across the blood-brain barrier in the adult mouse for central nervous system gene therapy*
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  72. DETERMINANTS OF AAV TRANSPORT ACROSS THE BLOOD-BRAIN BARRIER
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  73. Introduction
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  74. Adeno-associated virus-mediated gene therapy in central nervous system genetic disorders
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  75. Minimally invasive routes of AAV administration to treat GM1 gangliosidosis
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  77. Faculty Opinions recommendation of Adeno-associated virus vector as a platform for gene therapy delivery.
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  78. Can we prove the existence of God using quantitative biology ? Retracted
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