AAV9: a potential blood-brain barrier buster

The Blood-Brain Barrier in Health and Chronic Neurodegenerative Disorders

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Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

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Membrane-Associated Heparan Sulfate Proteoglycan Is a Receptor for Adeno-Associated Virus Type 2 Virions

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Retrograde Viral Delivery of IGF-1 Prolongs Survival in a Mouse ALS Model

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Recombinant AAV Viral Vectors Pseudotyped with Viral Capsids from Serotypes 1, 2, and 5 Display Differential Efficiency and Cell Tropism after Delivery to Different Regions of the Central Nervous System

Burger, C., Gorbatyuk, O. S., Velardo, M. J., Peden, C. S., Williams, P., Zolotukhin, S., Reier, P. J., Mandel, R. J., & Muzyczka, N. (2004) · Molecular Therapy · doi:10.1016/j.ymthe.2004.05.024 · Cited by 741 · see citing works

Parkinson-Like Neurodegeneration Induced by Targeted Overexpression of α-Synuclein in the Nigrostriatal System

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αVβ5 integrin: a co-receptor for adeno-associated virus type 2 infection

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From the Cover: Recombinant adeno-associated virus type 2, 4, and 5 vectors: Transduction of variant cell types and regions in the mammalian central nervous system

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The atomic structure of adeno-associated virus (AAV-2), a vector for human gene therapy

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Recombinant adeno-associated virus type 2, 4, and 5 vectors: Transduction of variant cell types and regions in the mammalian central nervous system

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Use of adeno-associated virus as a mammalian DNA cloning vector: transduction of neomycin resistance into mammalian tissue culture cells.

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Strategies to advance translational research into brain barriers

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Human fibroblast growth factor receptor 1 is a co-receptor for infection by adeno-associated virus 2

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Binding of Adeno-associated Virus Type 5 to 2,3-Linked Sialic Acid Is Required for Gene Transfer

Walters, R. W., Yi, S. M. P., Keshavjee, S. H., Brown, K. E., Welsh, M. J., Chiorini, J. A., & Zabner, J. (2001) · Journal of Biological Chemistry · doi:10.1074/jbc.m101559200 · Cited by 360 · see citing works

α2,3 and α2,6 N-Linked Sialic Acids Facilitate Efficient Binding and Transduction by Adeno-Associated Virus Types 1 and 6

Wu, Z., Miller, E. B., Agbandje‐McKenna, M., & Samulski, R. J. (2006) · Journal of Virology · doi:10.1128/jvi.00895-06 · Cited by 327 · see citing works

Engineering and Selection of Shuffled AAV Genomes: A New Strategy for Producing Targeted Biological Nanoparticles

Li, W., Asokan, A., Wu, Z., Van Dyke, T., DiPrimio, N., Johnson, J. S., Govindaswamy, L., Agbandje-McKenna, M., Leichtle, S., Eugene Redmond Jr, D., et al. (2008) · Molecular Therapy · doi:10.1038/mt.2008.100 · Cited by 261 · see citing works

Structure of Adeno-Associated Virus Serotype 8, a Gene Therapy Vector

Nam, H.-J., Lane, M. D., Padron, E., Gurda, B. L., McKENNA, R. V., Kohlbrenner, E., Aslanidi, G., Byrne, B. J., Muzyczka, N., Zolotukhin, S., et al. (2007) · Journal of Virology · doi:10.1128/jvi.01304-07 · Cited by 256 · see citing works

Single Amino Acid Changes Can Influence Titer, Heparin Binding, and Tissue Tropism in Different Adeno-Associated Virus Serotypes

Wu, Z., Asokan, A., Grieger, J. C., Lakshmanan Govindasamy, A., Agbandje‐McKenna, M., & Samulski, R. J. (2006) · Journal of Virology · doi:10.1128/jvi.01288-06 · Cited by 239 · see citing works

Adeno-associated virus 2 co-receptors?-first reply

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Long-term consequences of human alpha-synuclein overexpression in the primate ventral midbrain

Eslamboli, A., Romero‐Ramos, M., Bürger, C., Björklund, T., Muzyczka, N., Mandel, R. J., Baker, H. F., Ridley, R. M., & Kirik, D. (2007) · Brain · doi:10.1093/brain/awl382 · Cited by 227 · see citing works

Intraventricular Brain Injection of Adeno-Associated Virus Type 1 (AAV1) in Neonatal Mice Results in Complementary Patterns of Neuronal Transduction to AAV2 and Total Long-Term Correction of Storage Lesions in the Brains of β-Glucuronidase-Deficient Mice

Passini, M. A., Watson, D. J., Vite, C. H., Landsburg, D. J., Feigenbaum, A. L., & Wolfe, J. H. N. (2003) · Journal of Virology · doi:10.1128/jvi.77.12.7034-7040.2003 · Cited by 213 · see citing works

Adeno-Associated Virus Type 2 Contains an Integrin α5β1 Binding Domain Essential for Viral Cell Entry

Asokan, A., Hamra, J. B., Govindasamy, L., Agbandje-McKenna, M., & Samulski, R. J. (2006) · Journal of Virology · doi:10.1128/jvi.00843-06 · Cited by 183 · see citing works

BARRIER MECHANISMS IN THE BRAIN, II. IMMATURE BRAIN

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Intracranial Adeno-Associated Virus-Mediated Delivery of Anti-Pan Amyloid β, Amyloid β40, and Amyloid β42 Single-Chain Variable Fragments Attenuates Plaque Pathology in Amyloid Precursor Protein Mice

Levites, Y., Jansen, K. R., Smithson, L. A., Dakin, R. S., Holloway, V. M., Das, P., & Golde, T. E. (2006) · Journal of Neuroscience · doi:10.1523/jneurosci.2795-06.2006 · Cited by 128 · see citing works

Structure of Adeno-Associated Virus Type 4

Padron, E., Bowman, V. D., Kaludov, N. K., Lakshmanan Govindasamy, A., Levy, H. C., Nick, P., McKENNA, R. V., Muzyczka, N., Chiorini, J. A., Baker, T. S., et al. (2005) · Journal of Virology · doi:10.1128/jvi.79.8.5047-5058.2005 · Cited by 108 · see citing works

Combining growth factor and stem cell therapy for amyotrophic lateral sclerosis

Suzuki, M., & Svendsen, C. N. (2008) · Trends in Neurosciences · doi:10.1016/j.tins.2008.01.006 · Cited by 96 · see citing works

Neonatal Intraperitoneal or Intravenous Injections of Recombinant Adeno-Associated Virus Type 8 Transduce Dorsal Root Ganglia and Lower Motor Neurons

Foust, K. D., Poirier, A. E., Pacak, C. A., Mandel, R. J., & Flotte, T. R. (2007) · Human Gene Therapy · doi:10.1089/hum.2007.093 · Cited by 79 · see citing works

Production, purification and preliminary X-ray crystallographic studies of adeno-associated virus serotype 1

Miller, E. B., Gurda, B. L., Lakshmanan Govindasamy, A., McKENNA, R. V., Zolotukhin, S., Muzyczka, N., & Agbandje‐McKenna, M. (2006) · Acta Crystallographica Section F Structural Biology and Crystallization Communications · doi:10.1107/s1744309106048184 · Cited by 58 · see citing works

Production, purification, crystallization and preliminary X-ray structural studies of adeno-associated virus serotype 5

DiMattia, M. A., Lakshmanan Govindasamy, A., Levy, H. C., Gurda, B. L., Kalina, A., Kohlbrenner, E., Chiorini, J. A., McKENNA, R. V., Muzyczka, N., Zolotukhin, S., et al. (2005) · Acta Crystallographica Section F Structural Biology and Crystallization Communications · doi:10.1107/s1744309105028514 · Cited by 42 · see citing works

Recombinant Adeno-Associated Virus-Mediated Global Anterograde Delivery of Glial Cell Line-Derived Neurotrophic Factor to the Spinal Cord: Comparison of Rubrospinal and Corticospinal Tracts in the Rat

Foust, K. D., Flotte, T. R., Reier, P. J., & Mandel, R. J. (2007) · Human Gene Therapy · doi:10.1089/hum.2007.104 · Cited by 37 · see citing works

Production, purification and preliminary X-ray crystallographic studies of adeno-associated virus serotype 7

Quesada, O., Gurda, B. L., Lakshmanan Govindasamy, A., McKENNA, R. V., Kohlbrenner, E., Aslanidi, G., Zolotukhin, S., Muzyczka, N., & Agbandje‐McKenna, M. (2007) · Acta Crystallographica Section F Structural Biology and Crystallization Communications · doi:10.1107/s1744309107060289 · Cited by 20 · see citing works

Blood-Brain Barrier: From Physiology to Disease and Back

Sweeney, M. D., Zhao, Z., Montagne, A., Nelson, A. R., & Zlokovic, B. V. (2019) · Physiological Reviews · doi:10.1152/physrev.00050.2017 · Cited by 2,294 · see citing works

Adeno-Associated Virus Serotype 9 Transduction in the Central Nervous System of Nonhuman Primates

Samaranch, L., Salegio, E. A., San Sebastian, W., Kells, A. P., Foust, K. D., Bringas, J. R., Lamarre, C., Forsayeth, J., Kaspar, B. K., & Bankiewicz, K. S. (2012) · Human Gene Therapy · doi:10.1089/hum.2011.200 · Cited by 299 · see citing works

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

Yang, B., Li, S., Wang, H., Guo, Y., Gessler, D. J., Cao, C., Su, Q., Kramer, J., Zhong, L., Ahmed, S. S., et al. (2014) · Molecular Therapy · doi:10.1038/mt.2014.68 · Cited by 213 · see citing works

Targeted Delivery of Self-Complementary Adeno-Associated Virus Serotype 9 to the Brain, Using Magnetic Resonance Imaging-Guided Focused Ultrasound

Thévenot, E., Jordão, J. F., O’Reilly, M. A., Markham, K. A., Weng, Y., Foust, K. D., Kaspar, B. K., Hynynen, K., & Aubert, I. (2012) · Human Gene Therapy · doi:10.1089/hum.2012.013 · Cited by 202 · see citing works

Gene therapy for the CNS using AAVs: The impact of systemic delivery by AAV9

Saraiva, J., Nobre, R. J., & Pereira de Almeida, L. (2016) · Journal of Controlled Release · doi:10.1016/j.jconrel.2016.09.011 · Cited by 201 · see citing works

Robust spinal motor neuron transduction following intrathecal delivery of AAV9 in pigs

Federici, T., Taub, J. S., Baum, G. R., Gray, S. J., Grieger, J. C., Matthews, K. A., Handy, C. R., Passini, M. A., Samulski, R. J., & Boulis, N. M. (2011) · Gene Therapy · doi:10.1038/gt.2011.130 · Cited by 169 · see citing works

Virally mediated Kcnq1 gene replacement therapy in the immature scala media restores hearing in a mouse model of human Jervell and Lange‐Nielsen deafness syndrome

Chang, Q., Wang, J., Li, Q., Kim, Y., Zhou, B., Wang, Y., Li, H., & Lin, X. (2015) · EMBO Molecular Medicine · doi:10.15252/emmm.201404929 · Cited by 119 · see citing works

A Safe and Reliable Technique for CNS Delivery of AAV Vectors in the Cisterna Magna

Taghian, T., Marosfoi, M. G., Puri, A. S., Cataltepe, O. I., King, R. M., Diffie, E. B., Maguire, A. S., Martin, D. R., Fernau, D., Batista, A. R., et al. (2019) · Molecular Therapy · doi:10.1016/j.ymthe.2019.11.012 · Cited by 111 · see citing works

Widespread neuron-specific transgene expression in brain and spinal cord following synapsin promoter-driven AAV9 neonatal intracerebroventricular injection

McLean, J. R., Smith, G. A., Rocha, E. M., Hayes, M. A., Beagan, J. A., Hallett, P. J., & Ole Isacson, A. (2014) · Neuroscience Letters · doi:10.1016/j.neulet.2014.05.044 · Cited by 107 · see citing works

Impact of Insulin Degrading Enzyme and Neprilysin in Alzheimer’s Disease Biology: Characterization of Putative Cognates for Therapeutic Applications

Jha, N. K., Jha, S. K., Kumar, D., Kejriwal, N., Sharma, R., Ambasta, R. K., & Kumar, P. (2015) · Journal of Alzheimer s Disease · doi:10.3233/jad-150379 · Cited by 88 · see citing works

BBB-crossing adeno-associated virus vector: An excellent gene delivery tool for CNS disease treatment

Chen, W., Yao, S., Wan, J., Tian, Y., Huang, L., Wang, S., Akter, F., Wu, Y., Yao, Y., & Zhang, X. (2021) · Journal of Controlled Release · doi:10.1016/j.jconrel.2021.03.029 · Cited by 81 · see citing works

Sustained Correction of Motoneuron Histopathology Following Intramuscular Delivery of AAV in Pompe Mice

ElMallah, M. K., Falk, D. J., Nayak, S., Federico, R. A., Sandhu, M. S., Poirier, A. E., Byrne, B. J., & Fuller, D. D. (2013) · Molecular Therapy · doi:10.1038/mt.2013.282 · Cited by 82 · see citing works

Gene therapy for Mucopolysaccharidoses

Sawamoto, K., Chen, H.-H., Alméciga-Díaz, C. J., Mason, R. W., & Tomatsu, S. (2017) · Molecular Genetics and Metabolism · doi:10.1016/j.ymgme.2017.12.434 · Cited by 76 · see citing works

Adenoassociated virus serotype 9-mediated gene therapy for x-linked adrenoleukodystrophy

Gong, Y., Mu, D., Prabhakar, S., Moser, A., Musolino, P., Ren, J., Breakefield, X. O., Maguire, C. A., & Eichler, F. S. (2015) · Molecular Therapy · doi:10.1038/mt.2015.6 · Cited by 74 · see citing works

Systemic AAV9 gene therapy improves the lifespan of mice with Niemann-Pick disease, type C1

Chandler, R. J., Williams, I. M., Gibson, A. L., Davidson, C. D., Incao, A. A., Hubbard, B. T., Porter, F. D., Pavan, W. J., & Venditti, C. P. (2016) · Human Molecular Genetics · doi:10.1093/hmg/ddw367 · Cited by 74 · see citing works

Recent advances in gene therapy for neurodevelopmental disorders with epilepsy

Turner, T. J., Zourray, C., Schorge, S., & Lignani, G. (2021) · Journal of Neurochemistry · doi:10.1111/jnc.15168 · Cited by 71 · see citing works

Telomerase gene therapy ameliorates the effects of neurodegeneration associated to short telomeres in mice

Whittemore, K., Derevyanko, A., Martinez, P., Serrano, R., Pumarola, M., Bosch, F., & Blasco, M. A. (2019) · Aging · doi:10.18632/aging.101982 · Cited by 69 · see citing works

Therapeutic Approaches in Lysosomal Storage Diseases

Fernández-Pereira, C., Millán, B. S., Gallardo‐Gómez, M., Pérez-Márquez, T., Alves-Villar, M., Melcón-Crespo, C., Fernández-Martín, J., & Ortolano, S. (2021) · Biomolecules · doi:10.3390/biom11121775 · Cited by 66 · see citing works

Cas9-mediated replacement of expanded CAG repeats in a pig model of Huntington’s disease

Yan, S., Zheng, X., Lin, Y., Li, C., Liu, Z., Li, J., Tu, Z., Zhao, Y., Huang, C., Chen, Y., et al. (2023) · Nature Biomedical Engineering · doi:10.1038/s41551-023-01007-3 · Cited by 66 · see citing works

Adeno-Associated Virus Vector for Central Nervous System Gene Therapy

Zhu, D., Schieferecke, A. J., Lopez, P. A., & Schaffer, D. V. (2021) · Trends in Molecular Medicine · doi:10.1016/j.molmed.2021.03.010 · Cited by 56 · see citing works

Targeted Gene Delivery to the Enteric Nervous System Using AAV: A Comparison Across Serotypes and Capsid Mutants

Benskey, M. J., Kuhn, N. C., Galligan, J. J., Garcia, J., Boye, S. L., Hauswirth, W. W., Mueller, C., Boye, S. L., & Manfredsson, F. P. (2015) · Molecular Therapy · doi:10.1038/mt.2015.7 · Cited by 47 · see citing works

MRI-Guided Focused Ultrasound for Targeted Delivery of rAAV to the Brain

Noroozian, Z., Xhima, K., Huang, Y., Kaspar, B. K., Kügler, S., Hynynen, K., & Aubert, I. (2019) · Methods in molecular biology · doi:10.1007/978-1-4939-9139-6_10 · Cited by 48 · see citing works

Peptide Ligands Incorporated into the Threefold Spike Capsid Domain to Re-Direct Gene Transduction of AAV8 and AAV9 In Vivo

Michelfelder, S., Varadi, K., Raupp, C., Hunger, A., Körbelin, J., Pahrmann, C., Schrepfer, S., Müller, O. J., Kleinschmidt, J. A., & Trepel, M. (2011) · PLoS ONE · doi:10.1371/journal.pone.0023101 · Cited by 46 · see citing works

Retrograde Gene Delivery to Hypoglossal Motoneurons Using Adeno-Associated Virus Serotype 9

ElMallah, M. K., Falk, D. J., Lane, M. A., Conlon, T. J., Lee, K., Shafi, N. I., Reier, P. J., Byrne, B. J., & Fuller, D. D. (2012) · Human Gene Therapy Methods · doi:10.1089/hgtb.2012.009 · Cited by 45 · see citing works

Photoacoustic Neuroimaging - Perspectives on a Maturing Imaging Technique and its Applications in Neuroscience

Bodea, S., & Westmeyer, G. G. (2021) · Frontiers in Neuroscience · doi:10.3389/fnins.2021.655247 · Cited by 45 · see citing works

AAV9 transduction mediated by systemic delivery of vector via retro-orbital injection in newborn, neonatal and juvenile mice

Prabhakar, S., Lüle, S., da Hora, C. C., Breakefield, X. O., & Cheah, P.-S. (2021) · EXPERIMENTAL ANIMALS · doi:10.1538/expanim.20-0186 · Cited by 44 · see citing works

Positron emission tomography reporter gene strategy for use in the central nervous system

Haywood, T., Beinat, C., Gowrishankar, G., Patel, C. B., Alam, I. S., Murty, S., & Gambhir, S. S. (2019) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1901645116 · Cited by 43 · see citing works

Adeno‐associated virus (AAV)-based gene therapy for glioblastoma

Xu, X., Chen, W., Zhu, W., Chen, J., Ma, B., Ding, J., Wang, Z., Li, Y., Wang, Y., & Zhang, X. (2021) · Cancer Cell International · doi:10.1186/s12935-021-01776-4 · Cited by 41 · see citing works

AAV1 is the optimal viral vector for optogenetic experiments in pigeons (Columba livia)

Rook, N., Tuff, J. M., Isparta, S., Masseck, O. A., Herlitze, S., Güntürkün, O., & Pusch, R. (2021) · Communications Biology · doi:10.1038/s42003-020-01595-9 · Cited by 36 · see citing works

Recombinant Adeno-Associated Virus Vectors for Gene Therapy of the Central Nervous System: Delivery Routes and Clinical Aspects

Słyk, Ż., Stachowiak, N., & Małecki, M. (2024) · Biomedicines · doi:10.3390/biomedicines12071523 · Cited by 39 · see citing works

Structural basis of receptor usage by the engineered capsid AAV-PHP.eB

Jang, S., Shen, H. K., Ding, X., Miles, T. F., & Gradinaru, V. (2022) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2022.07.011 · Cited by 32 · see citing works

Adeno-Associated Viral Vectors as Versatile Tools for Parkinson’s Research, Both for Disease Modeling Purposes and for Therapeutic Uses

Fajardo-Serrano, A., Rico, A. J., Roda, E., Honrubia, A., Arrieta, S., Ariznabarreta, G., Chocarro, J., Lorenzo-Ramos, E., Pejenaute, A., Vázquez, A., et al. (2021) · International Journal of Molecular Sciences · doi:10.3390/ijms22126389 · Cited by 32 · see citing works

Innovative Therapeutic Approaches for Huntington’s Disease: From Nucleic Acids to GPCR-Targeting Small Molecules

Komatsu, H. (2021) · Frontiers in Cellular Neuroscience · doi:10.3389/fncel.2021.785703 · Cited by 32 · see citing works

Regulated protein expression for in vivo gene therapy for neurological disorders: Progress, strategies, and issues

Manfredsson, F. P., Bloom, D. C., & Mandel, R. J. (2012) · Neurobiology of Disease · doi:10.1016/j.nbd.2012.03.001 · Cited by 29 · see citing works

Cell-penetrating peptides enhance the transduction of adeno-associated virus serotype 9 in the central nervous system

Meng, Y., Sun, D., Qin, Y., Dong, X., Luo, G., & Liu, Y. (2021) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2021.02.019 · Cited by 29 · see citing works

Intravenous Delivery of Adeno-Associated Viral Vector Serotype 9 Mediates Effective Gene Expression in Ischemic Stroke Lesion and Brain Angiogenic Foci

Shen, F., Kuo, R., Milon-Camus, M., Han, Z., Jiang, L., Young, W. L., & Su, H. (2012) · Stroke · doi:10.1161/strokeaha.112.662965 · Cited by 26 · see citing works

In vivo gene editing via homology-independent targeted integration for adrenoleukodystrophy treatment

Hong, S.-A., Seo, J. H., Wi, S., Jung, E. S., Yu, J., Hwang, G., Yu, J. H., Baek, A., Park, S., Bae, S., et al. (2021) · Molecular Therapy · doi:10.1016/j.ymthe.2021.05.022 · Cited by 26 · see citing works

AAV-mediated siRNA against TRPV1 reduces nociception in a rat model of bone cancer pain

Zhang, S., Zhao, J., & Meng, Q. (2019) · Neurological Research · doi:10.1080/01616412.2019.1639317 · Cited by 26 · see citing works

Over the barrier and through the blood: To CNS delivery we go

Foust, K. D., & Kaspar, B. K. (2009) · Cell Cycle · doi:10.4161/cc.8.24.10245 · Cited by 25 · see citing works

Systemic Correction of Murine Glycogen Storage Disease Type IV by an AAV-Mediated Gene Therapy

Yi, H., Zhang, Q., Brooks, E. D., Yang, C., Thurberg, B. L., Kishnani, P. S., & Sun, B. (2016) · Human Gene Therapy · doi:10.1089/hum.2016.099 · Cited by 24 · see citing works

Preclinical Development of New Therapy for Glycogen Storage Diseases

Sun, B., Brooks, E. D., & Koeberl, D. D. (2015) · Current Gene Therapy · doi:10.2174/1566523215666150630132253 · Cited by 24 · see citing works

Candidate Diseases for Prenatal Gene Therapy

David, A. L., & Waddington, S. N. (2012) · Humana Press eBooks · doi:10.1007/978-1-61779-873-3_2 · Cited by 24 · see citing works

Development of an AAV9 coding for a 3XFLAG-TALEfrat#8-VP64 able to increase in vivo the human frataxin in YG8R mice

Chapdelaine, P., Gérard, C., Sanchez, N., Cherif, K., Rousseau, J., Ouellet, D. L., Jauvin, D., & Tremblay, J. P. (2016) · Gene Therapy · doi:10.1038/gt.2016.36 · Cited by 21 · see citing works

AAV9: Over the Fence and Into the Woods …

Forsayeth, J., & Bankiewicz, K. S. (2011) · Molecular Therapy · doi:10.1038/mt.2011.95 · Cited by 19 · see citing works

Development of Engineered-U1 snRNA Therapies: Current Status

Gonçalves, M., Santos, J. I., Coutinho, M. F., Matos, L., & Alves, S. (2023) · International Journal of Molecular Sciences · doi:10.3390/ijms241914617 · Cited by 19 · see citing works

Increased Frataxin Expression Induced in Friedreich Ataxia Cells by Platinum TALE-VP64s or Platinum TALE-SunTag

Cherif, K., Gérard, C., Rousseau, J., Ouellet, D. L., Chapdelaine, P., & Tremblay, J. P. (2018) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2018.04.009 · Cited by 18 · see citing works

PET imaging of focused-ultrasound enhanced delivery of AAVs into the murine brain

Ajenjo, J., Seo, J. W., Foiret, J. L., Wu, B., Raie, M. N., Wang, J., Fite, B. Z., Zhang, N., Malek, R. S., Beinat, C., et al. (2023) · Theranostics · doi:10.7150/thno.85549 · Cited by 14 · see citing works

Reversibility and therapeutic development for neurodevelopmental disorders, insights from genetic animal models

Megagiannis, P., Suresh, R., Rouleau, G. A., & Zhou, Y. (2022) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2022.114562 · Cited by 12 · see citing works

In utero gene transfer to the mouse nervous system

Rahim, A. A., Wong, A. M. S., Buckley, S. M. K., Chan, J. K. Y., David, A. L., Cooper, J. D., Coutelle, C. C., Peebles, D., & Waddington, S. N. (2010) · Biochemical Society Transactions · doi:10.1042/bst0381489 · Cited by 11 · see citing works

Is RNA manipulation a viable therapy for spinal muscular atrophy?

Horne, C., & Young, P. J. (2009) · Journal of the Neurological Sciences · doi:10.1016/j.jns.2009.08.055 · Cited by 11 · see citing works

Sequential Adeno-Associated Viral Vector Serotype 9–Green Fluorescent Protein Gene Transfer Causes Massive Inflammation and Intense Immune Response in Rat Striatum

Yang, C., Hao, F., He, J., Lu, T., Klein, R. L., Zhao, L., & Duan, W. (2016) · Human Gene Therapy · doi:10.1089/hum.2015.083 · Cited by 11 · see citing works

Adeno-associated virus vector hydrogel formulations for brain cancer gene therapy applications

Słyk, Ż., Wrzesień, R., Barszcz, S., Gawrychowski, K., & Małecki, M. (2023) · Biomedicine & Pharmacotherapy · doi:10.1016/j.biopha.2023.116061 · Cited by 10 · see citing works

Histochemical localization of palmitoyl protein thioesterase-1 activity

Dearborn, J. T., Ramachandran, S., Shyng, C., Lu, J.-Y., Thornton, J., Hofmann, S. L., & Sands, M. S. (2015) · Molecular Genetics and Metabolism · doi:10.1016/j.ymgme.2015.11.004 · Cited by 10 · see citing works

Gene Therapy for the Treatment of Parkinson’s Disease: The Nature of the Biologics Expands the Future Indications

Fiandaca, M. S., Bankiewicz, K. S., & Federoff, H. J. (2012) · Pharmaceuticals · doi:10.3390/ph5060553 · Cited by 10 · see citing works

The development of flexible lentiviral vectors for gene transfer in the CNS

Manfredsson, F. P., & Mandel, R. J. (2011) · Experimental Neurology · doi:10.1016/j.expneurol.2011.03.013 · Cited by 9 · see citing works

Challenges for Gene Therapy of CNS Disorders and Implications for Parkinson's Disease Therapies

Broadstock, M., & Yáñez‐Muñoz, R. J. (2012) · Human Gene Therapy · doi:10.1089/hum.2012.2507 · Cited by 9 · see citing works

Use of adeno-associated viruses for transgenic modulation of microglia structure and function: A review of technical considerations and challenges

Ball, J. B., Frank, M. G., Green‐Fulgham, S. M., & Watkins, L. R. (2024) · Brain Behavior and Immunity · doi:10.1016/j.bbi.2024.03.005 · Cited by 9 · see citing works

Syndecan-4 Mediates the Cellular Entry of Adeno-Associated Virus 9

Hudák, A., Roach, M., Pusztai, D., Pettkó‐Szandtner, A., Letoha, A., Szilák, L., Azzouz, M., & Letoha, T. (2023) · International Journal of Molecular Sciences · doi:10.3390/ijms24043141 · Cited by 8 · see citing works

Protein LY6E as a candidate for mediating transport of adeno-associated virus across the human blood-brain barrier

Ille, A. M., Kishel, E., Bodea, R., Ille, A. L., Lamont, H., & Amico-Ruvio, S. A. (2020) · Journal of NeuroVirology · doi:10.1007/s13365-020-00890-9 · Cited by 8 · see citing works

An Induced Pluripotent Stem Cell-Derived Human Blood–Brain Barrier (BBB) Model to Test the Crossing by Adeno-Associated Virus (AAV) Vectors and Antisense Oligonucleotides

Selvakumaran, J., Ursu, S., Bowerman, M., Lu‐Nguyen, N., Wood, M. J. A., Malerba, A., & Yáñez‐Muñoz, R. J. (2023) · Biomedicines · doi:10.3390/biomedicines11102700 · Cited by 7 · see citing works

Gene editing for collagen disorders: current advances and future perspectives

Kocsy, K., Wilkinson, H., Felix-Ilemhenbhio, F., Bax, B. D., Agtmael, T. V., Azzouz, M., & Majid, A. (2025) · Gene Therapy · doi:10.1038/s41434-025-00560-7 · Cited by 6 · see citing works

Mechanistic Evaluation of miRNAs and Their Targeted Genes in the Pathogenesis and Therapeutics of Parkinson’s Disease

Arora, T., Sharma, G., Prashar, V., Singh, R., Sharma, A., Changotra, H., & Parkash, J. (2024) · Molecular Neurobiology · doi:10.1007/s12035-024-04261-x · Cited by 6 · see citing works

Treatment of glutaric aciduria type I (GA-I) via intracerebroventricular delivery of GCDH

Guo, L., Li, Z., Li, Y., Qu, B., Jiao, G., Chen, L., Lu, Z., Wang, X.-G., Huang, C., Du, H., et al. (2022) · Fundamental Research · doi:10.1016/j.fmre.2022.08.013 · Cited by 5 · see citing works

Reprogramming Adeno-Associated Virus Tropism Via Displayed Peptides Tiling Receptor-LigandsPreprint

Portell, A. J., Ford, K. M., Suhardjo, A., Rainaldi, J., Bublik, M. N., Sanghvi, M., Kumar, A., Wing, M. K., Palmer, N. D., Le, D. A., et al. (2022) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2022.09.26.509383 · Cited by 5 · see citing works

Arming AAV9 with a Single-Chain Fragment Variable Antibody Against PD-1 for Systemic Glioblastoma Therapy

Maksoud, S., Schweiger, M. W., Tabet, E. I., Xiao, T., Hokayem, J. E., Zinter, M., de Carvalho, L. A., Breyne, K., Noske, D. P., Chiocca, E. A., et al. (2024) · Molecular Neurobiology · doi:10.1007/s12035-024-04406-y · Cited by 4 · see citing works

Novel Approaches to Deliver Molecular Therapeutics in Cardiac Disease Using Adeno-Associated Virus Vectors

Rapti, K., Hajjar, R. J., & Weber, T. (2012) · Humana Press eBooks · doi:10.1007/978-1-61779-891-7_12 · Cited by 4 · see citing works

PET imaging of AAV9 and AAVBR1 trafficking in normal mice

Bansal, A., Sharma, S., Kethamreddy, M., & Pandey, M. K. (2025) · Scientific Reports · doi:10.1038/s41598-025-86815-0 · Cited by 3 · see citing works

Ten years of disease-modifying therapy in spinal muscular atrophy: lessons learned and future directions

Mercuri, E. M., Finkel, R. S., & Francesco Muntoni, A. (2026) · Nature Reviews Neurology · doi:10.1038/s41582-026-01224-9 · Cited by 3 · see citing works

Effects of chemogenetic inhibition of serotonergic raphe-striatal neurons on L-DOPA-induced behaviors and neurochemistry in a rat model of Parkinson’s disease

Budrow, C., Bernal-Conde, L. D., Sandoval, I. M., Galfano, A., Grezenko, H., Velazquez, A., Marmion, D. J., Flores-Barrera, E., Venkatesh, S., Coyle, M., et al. (2026) · Acta Neuropathologica Communications · doi:10.1186/s40478-026-02221-1 · Cited by 2 · see citing works

Optimization of systemic AAV9 gene therapy in Niemann–Pick disease, type C1 mice

Mylvara, A. V., Gibson, A. L., Gu, T., Davidson, C. D., Incao, A. A., Melnyk, K. D., Gembic, S., Pierre-Jacques, D., Cologna, S. M., Venditti, C. P., et al. (2026) · Life Science Alliance · doi:10.26508/lsa.202402874 · Cited by 2 · see citing works

Treatment and Management of Autosomal Recessive Cerebellar Ataxias: Current Advances and Future Perspectives

Dupré, N., Salem, I. H., Beaudin, M., & Klein, C. J. (2022) · CNS & Neurological Disorders - Drug Targets · doi:10.2174/1871527321666220418114846 · Cited by 2 · see citing works

Gene therapy targeting the blood-brain barrier

Körbelin, J., Pereira, A. A., & Schwaninger, M. (2024) · Vitamins and hormones · doi:10.1016/bs.vh.2024.03.001 · Cited by 2 · see citing works

Long‐Term Safety and Efficacy of AAV9 Vectors Expressing Human SMN1 Gene: A Preclinical Study

Mansouri, V., Bemanalizadeh, M., Ahmadbeigi, N., Shakeri, R., & Saffar, H. (2025) · Basic & Clinical Pharmacology & Toxicology · doi:10.1111/bcpt.70064 · Cited by 2 · see citing works

Perinatal Gene Therapy

Shangaris, P., & David, A. L. (2016) · Stem cell biology and regenerative medicine · doi:10.1007/978-1-4939-3483-6_19 · Cited by 2 · see citing works

Development of viral & non-viral episomal vectors for gene therapy applications

Kymäläinen, H. (2012) · OpenGrey (Institut de l'Information Scientifique et Technique) · Cited by 2 · see citing works

Optimization of systemic AAV9 gene therapy in Niemann-Pick disease type C1 micePreprint

Mylvara, A. V., Gibson, A. L., Gu, T., Davidson, C. D., Incao, A. A., Melnyk, K. D., Gembic, S. R., Pierre-Jacques, D., Cologna, S. M., Venditti, C. P., et al. (2024) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2024.06.07.597901 · Cited by 2 · see citing works

Targeted Gene Therapy for Ischemic Stroke

Shen, F., & Su, H. (2015) · Neuromethods · doi:10.1007/978-1-4939-2306-9_8 · Cited by 1 · see citing works

Viral and non-viral cellular therapies for neurodegeneration

Srivastav, J., & Sharma, S. (2026) · Frontiers in Medicine · doi:10.3389/fmed.2025.1718669 · Cited by 1 · see citing works

Gene Therapy for Parkinson’s Disease: Towards Non Invasive Approaches

Rodrı́guez, M., Jose, M., & Castro, R. (2011) · InTech eBooks · doi:10.5772/20631 · Cited by 1 · see citing works

Terapia génica no invasiva en enfermedades neurológicas

Armas, J. M. B., Castro‐Hernández, J., Díaz, M. R., & Fuentes, R. C. (2011) · Revista de Neurología · doi:10.33588/rn.5210.2010604 · Cited by 1 · see citing works

Viral Vectors and Other Modulatory Biologics

Mader, B. J., & Boulis, N. M. (2017) · Elsevier eBooks · doi:10.1016/b978-0-12-800454-8.00009-4 · Cited by 1 · see citing works

Viral‐Mediated Delivery of shRNA and miRNA

Manfredsson, F. P. (2013) · Wiley eBooks · doi:10.1002/9781118610749.ch11 · Cited by 1 · see citing works

Gene therapy

Mehta, V., & David, A. L. (2012) · Cambridge University Press eBooks · doi:10.1017/cbo9780511997778.043

GLT-1 over-expression attenuates visceral nociception by pharmacological and gene therapy approaches

Roman, K. (2012) · OhioLink ETD Center (Ohio Library and Information Network)

IN UTERO GENE DELIVERY OF AAV VECTORS FOR EFFICIENT TREATMENT OF MUSCLE DISORDERS

Koppanati, B. M. (2010) · D-Scholarship@Pitt (University of Pittsburgh)

Brain gene therapy: triumph and future of nanotechnology

Castro, R. (2012)

Intravenously administered gene therapy for neuronopathic Gaucher disease

Massaro, G. (2018) · UCL Discovery (University College London)

Gene Therapy: Principles and Clinical Potential

Buckley, S. M. K., & David, A. L. (2019) · Cambridge University Press eBooks · doi:10.1017/9781108564434.053

Intrathecal Vector Delivery in Juvenile Rats via Lumbar Cistern Injection

Donsante, A., Rasmussen, S. A., & Fridovich‐Keil, J. L. (2024) · Journal of Visualized Experiments · doi:10.3791/66463

CRISPR/Cas9-based genetic engineering for translational research in neurological disorders

Megagiannis, P., Rouleau, G. A., & Zhou, Y. (2024) · Elsevier eBooks · doi:10.1016/b978-0-443-19041-4.00021-2

Determining recombinant AAV capsid extracellular and intracellular biodistribution by dual radioisotope labeling

Chandler, R. J. (2024) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2024.101373

Systemic delivery of AAV-GFM1 corrects COXPD1 molecular alterations in Gfm1R671C/− mice

Molina‐Berenguer, M., Herrero-Martínez, D., Vallbona-Garcia, A., Vila‐Julià, F., Cámara, Y., Vales, Á., González‐Aseguinolaza, G., Torres‐Torronteras, J., & Martí, R. (2026) · EMBO Molecular Medicine · doi:10.1038/s44321-026-00426-4

rAAV-Mediated Gene Transfer For Study of Pathological Mechanisms and Therapeutic Intervention in Canavan's Disease: A Dissertation

Ahmed, S. S. (2014) · eScholarship@UMassChan · doi:10.13028/m25s3r

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

Zhang, H., Yang, B., Mu, X., Ahmed, S. S., Su, Q., He, R., Wang, H., Mueller, C., Sena-Esteves, M., Brown, R., et al. (2011) · Molecular Therapy · doi:10.1038/mt.2011.98 · Cited by 291 · see citing works

In This Issue

(2011) · Molecular Therapy · doi:10.1038/mt.2011.148

Taking a Hint from Structural Biology: To Better Understand AAV Transport across the BBB

Wang, D., & Gao, G. (2018) · Molecular Therapy · doi:10.1016/j.ymthe.2018.01.005 · Cited by 4 · see citing works

Recombinant AAV-mediated gene delivery to the central nervous system

Tenenbaum, L., Chtarto, A., Lehtonen, E., Velu, T. J., Brotchi, J., & Levivier, M. (2004) · The Journal of Gene Medicine · doi:10.1002/jgm.506 · Cited by 204 · see citing works

724. A Comprehensive Map of CNS Transduction by Eight Recombinant Adeno-Associated Virus Serotypes upon Cerebrospinal Fluid Administration in Pigs

Sorrentino, N. C., Maffia, V., Strollo, S., Cacace, V., Romagnoli, N., Manfredi, A., Ventrella, D., Dondi, F., Barone, F., Giunti, M., et al. (2016) · Molecular Therapy · doi:10.1016/s1525-0016(16)33532-8