Cited by 208Virus-based gene therapy researchRNA Interference and Gene DeliveryBarrier Structure and Function Studies

The DOI matches its Crossref record; 28 of the 29 references it cites were found in a registry or an index.

AAV8, 9, Rh10, Rh43 Vector Gene Transfer in the Rat Brain: Effects of Serotype, Promoter and Purification Method

Open access

Klein, Ronald L, Dayton, Robert D, Tatom, Jason B, Henderson, Karen M, Henning, Phillip P·2008·Molecular Therapy

Open this paper in CoChat — free account Open PDF

Abstract

We compared adeno-associated virus (AAV) serotypes for expression levels of green fluorescent protein (GFP) in the adult rat hippocampus by biophotonic imaging. Preparations of AAV serotypes 8, 9, Rh10, and Rh43 incorporating cytomegalovirus (CMV) promoter-driven GFP were purified by a CsCl method. Neither AAV Rh10 nor AAV Rh43 produced greater levels of GFP than AAV8, which was used as a reference. For AAV9, there was an increase relative to AAV8. The CsCl-purified AAV8 displayed an astroglial transduction pattern in contrast to the expected neuronal expression of other AAVs. After preparing the same CMV-GFP plasmid in AAV8 with an iodixanol purification method, the expected neuronal pattern resulted. The astroglial expression with the CsCl AAV8 was probably due to relatively high levels of protein impurities. We compared the CMV promoter with the CMV/chicken beta-actin (CBA) promoter in the context of AAV8, both prepared by iodixanol, and found the CBA promoter to produce stronger GFP expression. At two doses of vectors optimized for serotype, promoter and purification, we did not observe serotype differences among AAV8, AAV9, or AAV Rh10. The purification method can therefore impact the transduction pattern as well as the results when comparing serotype strengths.

Integrity signals

Registry

Crossref · 10.1038/sj.mt.6300331

Cites

28 of 28 references verified0 citation errors · 0 not checkable

Statistics

Recomputing p-values needs the paper’s full text.

Claims the paper makes

We couldn’t retrieve this paper’s full text, so its claims and self-check weren’t run. The registry and citation checks still apply.

Claims other papers make about this one

Each is checked against this paper’s own text: does it say what the citing paper says it does?

The rat brain in stereotaxic coordinates
Palkovits, M. (1983) · Neuropeptides · doi:10.1016/0143-4179(83)90049-5 · Cited by 28,959
The Rat Brain in Stereotaxic Coordinates
Paxinos, G., & Watson, C. (1983) · Elsevier eBooks · doi:10.1016/c2009-0-63235-9 · Cited by 28,585
Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy
Gao, G.-P., Alvira, M. R., Wang, L., Calcedo, R., Johnston, J., & Wilson, J. M. (2002) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.182412299 · Cited by 1,520
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
Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart
Wang, Z., Zhu, T., Qiao, C., Zhou, L., Wang, B., Zhang, J., Chen, C., Li, J., & Xiao, X. (2005) · Nature Biotechnology · doi:10.1038/nbt1073 · Cited by 622
Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8
Inagaki, K., Fuess, S., Storm, T. A., Gibson, G. A., Mctiernan, C. F., Kay, M. A., & Nakai, H. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2006.03.014 · Cited by 610
Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors
Zolotukhin, S., Potter, M., Zolotukhin, I., Sakai, Y., Loiler, S., Fraites, T. J., Chiodo, V. A., Philipsberg, T., Muzyczka, N., Hauswirth, W. W., et al. (2002) · Methods · doi:10.1016/s1046-2023(02)00220-7 · Cited by 564
Recombinant adeno-associated virus type 2, 4, and 5 vectors: Transduction of variant cell types and regions in the mammalian central nervous system
Davidson, B. L., Stein, C. S., Heth, J. A., Martins, I., Kotin, R. M., Derksen, T. A., Zabner, J., Ghodsi, A., & Chiorini, J. A. (2000) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.97.7.3428 · Cited by 531
Contribution of glial cells to the development of amyloid plaques in Alzheimer’s disease
Nagele, R. G., Węgiel, J., Venkataraman, V., Imaki, H., Wang, K.-C., & Wegiel, J. (2004) · Neurobiology of Aging · doi:10.1016/j.neurobiolaging.2004.01.007 · Cited by 522
New recombinant serotypes of AAV vectors
Gao, G., Vandenberghe, L., & Wilson, J. (2005) · Current Gene Therapy · doi:10.2174/1566523054065057 · Cited by 487

Show all 29 references

Sorted by citations

Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection
Zincarelli, C., Soltys, S., Rengo, G., & Rabinowitz, J. E. (2008) · Molecular Therapy · doi:10.1038/mt.2008.76 · Cited by 1,490
Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes
Foust, K. D., Nurre, E., Montgomery, C. L., Hernandez, A., Chan, C. M., & Kaspar, B. K. (2009) · Nature Biotechnology · doi:10.1038/nbt.1515 · Cited by 1,456
Various AAV Serotypes and Their Applications in Gene Therapy: An Overview
Issa, S. S., Shaimardanova, A. A., Solovyeva, V. V., & Rizvanov, A. A. (2023) · Cells · doi:10.3390/cells12050785 · Cited by 339
Gene therapy for neurological disorders: progress and prospects
Deverman, B. E., Ravina, B. M., Bankiewicz, K. S., Paul, S. M., & Sah, D. W. Y. (2018) · Nature Reviews Drug Discovery · doi:10.1038/nrd.2018.110 · Cited by 306
Comparative Analysis of Adeno-Associated Viral Vector Serotypes 1, 2, 5, 7, And 8 in Mouse Brain
Taymans, J.-M., Vandenberghe, L. H., Haute, C. V. D., Thiry, I., Deroose, C. M., Mortelmans, L., Wilson, J. M., Debyser, Z., & Baekelandt, V. (2007) · Human Gene Therapy · doi:10.1089/hum.2006.178 · Cited by 305
Comparative analyses of adeno-associated viral vector serotypes 1, 2, 5, 8 and 9 in marmoset, mouse and macaque cerebral cortex
Watakabe, A., Ohtsuka, M., Kinoshita, M., Takaji, M., Isa, K., Mizukami, H., Ozawa, K., Isa, T., & Yamamori, T. (2015) · Neuroscience Research · doi:10.1016/j.neures.2014.09.002 · Cited by 303
Adeno-Associated Virus-Based Gene Therapy for CNS Diseases
Hocquemiller, M., Giersch, L., Audrain, M., Parker, S., & Cartier, N. (2016) · Human Gene Therapy · doi:10.1089/hum.2016.087 · Cited by 291
A survey of ex vivo/in vitro transduction efficiency of mammalian primary cells and cell lines with Nine natural adeno-associated virus (AAV1-9) and one engineered adeno-associated virus serotype
Ellis, B. L., Hirsch, M. L., Barker, J. C., Connelly, J. P., Steininger, R. J., & Porteus, M. H. (2013) · Virology Journal · doi:10.1186/1743-422x-10-74 · Cited by 252
Erratum: Gene therapy for neurological disorders: progress and prospects
Deverman, B. E., Ravina, B. M., Bankiewicz, K. S., Paul, S. M., & Sah, D. W. Y. (2018) · Nature Reviews Drug Discovery · doi:10.1038/nrd.2018.158 · Cited by 248
Structural insight into the unique properties of adeno-associated virus serotype 9
DiMattia, M. A., Nam, H.-J., Van Vliet, K., Mitchell, M., Bennett, A., Gurda, B. L., McKenna, R., Olson, N. H., Sinkovits, R. S., Potter, M., et al. (2012) · Journal of Virology · doi:10.1128/jvi.07232-11 · Cited by 219
Adeno-Associated Virus Technologies and Methods for Targeted Neuronal Manipulation
Haery, L., Deverman, B. E., Matho, K. S., Cetin, A. H., Woodard, K., Cepko, C. L., Guerin, K. I., Rego, M. A., Ersing, I., Bächle, S. M., et al. (2019) · Frontiers in Neuroanatomy · doi:10.3389/fnana.2019.00093 · Cited by 220
Capsid Serotype and Timing of Injection Determines AAV Transduction in the Neonatal Mice Brain
Chakrabarty, P., Rosario, A. M., Cruz, P. E., Siemienski, Z. B., Ceballos‐Diaz, C., Crosby, K. W., Jansen, K. R., Borchelt, D., Kim, J., Jankowsky, J. L., et al. (2013) · PLoS ONE · doi:10.1371/journal.pone.0067680 · Cited by 212
Molecular evolution of adeno-associated virus for enhanced glial gene delivery
Koerber, J. T., Klimczak, R., Jang, J.-H., Dalkara, D., Flannery, J. G., & Schaffer, D. V. (2009) · Molecular Therapy · doi:10.1038/mt.2009.184 · Cited by 181
Role of Phosphatidylinositol Clathrin Assembly Lymphoid-Myeloid Leukemia (PICALM) in Intracellular Amyloid Precursor Protein (APP) Processing and Amyloid Plaque Pathogenesis
Xiao, Q., Gil, S.-C., Yan, P., Wang, Y., Han, S., Gonzales, E., Perez, R., Cirrito, J. R., & Lee, J.-M. (2012) · Journal of Biological Chemistry · doi:10.1074/jbc.m111.338376 · Cited by 166
Parkinson's Disease Gene Therapy: Success by Design Meets Failure by Efficacy
Bartus, R. T., Weinberg, M. S., & Samulski, R. J. (2013) · Molecular Therapy · doi:10.1038/mt.2013.281 · Cited by 171
Efficient gene delivery and selective transduction of glial cells in the mammalian brain by AAV serotypes isolated from nonhuman primates
Lawlor, P. A., Bland, R. J., Mouravlev, A., Young, D., & During, M. J. (2009) · Molecular Therapy · doi:10.1038/mt.2009.170 · Cited by 161
Axonal transport of adeno-associated viral vectors is serotype-dependent
Salegio, E. A., Samaranch, L., Kells, A. P., Mittermeyer, G., San Sebastian, W., Zhou, S., Beyer, J., Forsayeth, J., & Bankiewicz, K. S. (2013) · Gene Therapy · doi:10.1038/gt.2012.27 · Cited by 156
Adeno-Associated Virus Serotypes 1, 8, and 9 Share Conserved Mechanisms for Anterograde and Retrograde Axonal Transport
Castle, M. J., Gershenson, Z. T., Giles, A. R., Holzbaur, E. L. F., & Wolfe, J. H. N. (2014) · Human Gene Therapy · doi:10.1089/hum.2013.189 · Cited by 165
Adeno-associated virus (AAV) gene therapy for neurological disease
Weinberg, M. S., Samulski, R. J., & McCown, T. J. (2012) · Neuropharmacology · doi:10.1016/j.neuropharm.2012.03.004 · Cited by 161
Controlling AAV Tropism in the Nervous System with Natural and Engineered Capsids
Castle, M. J., Turunen, H. T., Vandenberghe, L. H., & Wolfe, J. H. (2016) · Methods in Molecular Biology · doi:10.1007/978-1-4939-3271-9_10 · Cited by 151
“Within the brain, AAV9 is most frequently observed to undergo axonal transport, and is the recommended choice if distal transduction is desired [21, 23, 34, 36, 57, 60].”
Background
Comparative Analysis of Cesium Chloride- and Iodixanol-Based Purification of Recombinant Adeno-Associated Viral Vectors for Preclinical Applications
Strobel, B., Miller, F., Rist, W., & Lamla, T. (2015) · Human Gene Therapy Methods · doi:10.1089/hgtb.2015.051 · Cited by 148
The advent of AAV9 expands applications for brain and spinal cord gene delivery
Dayton, R. D., Wang, D. B., & Klein, R. L. (2012) · Expert Opinion on Biological Therapy · doi:10.1517/14712598.2012.681463 · Cited by 144
Self-citation
Glial promoter selectivity following AAV-delivery to the immature brain
von Jonquieres, G., Mersmann, N., Klugmann, C. B., Harasta, A. E., Lutz, B., Teahan, O., Housley, G. D., Fröhlich, D., Krämer-Albers, E.-M., & Klugmann, M. (2013) · PLoS ONE · doi:10.1371/journal.pone.0065646 · Cited by 133
Rapid and simplified purification of recombinant adeno-associated virus
Guo, P., El‐Gohary, Y., Prasadan, K. L., Shiota, C., Xiao, X., Wiersch, J. C., Paredes, J. L., Tulachan, S., & Gittes, G. K. (2012) · Journal of Virological Methods · doi:10.1016/j.jviromet.2012.04.004 · Cited by 130
Efficient transduction of non-human primate motor neurons after intramuscular delivery of recombinant AAV serotype 6
Towne, C., Schneider, B. L., Kieran, D., Redmond, D. E., & Aebischer, P. (2010) · Gene Therapy · doi:10.1038/gt.2009.119 · Cited by 124
Spreading of α-Synuclein and Tau: A Systematic Comparison of the Mechanisms Involved
Vasili, E., Dominguez‐Meijide, A., & Outeiro, T. F. (2019) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2019.00107 · Cited by 121
A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency
Tordo, J., O’Leary, C., Antunes, A. S. L. M., Palomar, N., Aldrin-Kirk, P., Basche, M., Bennett, A., D’Souza, Z., Gleitz, H., Godwin, A., et al. (2018) · Brain · doi:10.1093/brain/awy126 · Cited by 113
Fractalkine overexpression suppresses tau pathology in a mouse model of tauopathy
Nash, K. R., Lee, D. C., Hunt, J. B., Morganti, J. M., Selenica, M.-L., Moran, P., Reid, P., Brownlow, M., Guang-Yu Yang, C., Savalia, M., et al. (2013) · Neurobiology of Aging · doi:10.1016/j.neurobiolaging.2012.12.011 · Cited by 108
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 112
Clinical Gene Therapy for Neurodegenerative Diseases: Past, Present, and Future
Piguet, F., Alves, S., & Cartier, N. (2017) · Human Gene Therapy · doi:10.1089/hum.2017.160 · Cited by 115
Fractalkine Over Expression Suppresses α-Synuclein-mediated Neurodegeneration
Nash, K. R., Moran, P., Finneran, D. J., Hudson, C., Robinson, J., Morgan, D., & Bickford, P. C. (2014) · Molecular Therapy · doi:10.1038/mt.2014.175 · Cited by 107
AAV Targeting of Glial Cell Types in the Central and Peripheral Nervous System and Relevance to Human Gene Therapy
O’Carroll, S. J., Cook, W. H., & Young, D. (2021) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2020.618020 · Cited by 112
CRISPR based editing of SIV proviral DNA in ART treated non-human primates
Mancuso, P., Chen, C., Kaminski, R., Gordon, J., Liao, S., Robinson, J. A., Smith, M. D., Liu, H., Sariyer, I. K., Sariyer, R., et al. (2020) · Nature Communications · doi:10.1038/s41467-020-19821-7 · Cited by 105
Systemic AAV9 gene transfer in adult GM1 gangliosidosis mice reduces lysosomal storage in CNS and extends lifespan
Weismann, C. M., Ferreira, J. S., Keeler, A. M., Su, Q., Qui, L., Shaffer, S. A., Xu, Z., Gao, G., & Sena‐Esteves, M. (2015) · Human Molecular Genetics · doi:10.1093/hmg/ddv168 · Cited by 102
Systemic AAVrh10 provides higher transgene expression than AAV9 in the brain and the spinal cord of neonatal mice
Tanguy, Y., Biferi, M. G., Besse, A., Astord, S., Cohen-Tannoudji, M., Marais, T., & Barkats, M. (2015) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2015.00036 · Cited by 103
Crtc1 Activates a Transcriptional Program Deregulated at Early Alzheimer's Disease-Related Stages
Parra‐Damas, A., Valero, J., Chen, M., España, J., Martín, E., Ferrer, I., Rodrı́guez-Álvarez, J., & Saura, C. A. (2014) · Journal of Neuroscience · doi:10.1523/jneurosci.5288-13.2014 · Cited by 104
Characterization of a novel adeno-associated viral vector with preferential oligodendrocyte tropism
Powell, S. K., Khan, N., Parker, C. L., Samulski, R. J., Matsushima, G., Gray, S. J., & McCown, T. J. (2016) · Gene Therapy · doi:10.1038/gt.2016.62 · Cited by 92
Correction of Brain Oligodendrocytes by AAVrh.10 Intracerebral Gene Therapy in Metachromatic Leukodystrophy Mice
Piguet, F., Sondhi, D., Piraud, M., Fouquet, F., Hackett, N. R., Ahouansou, O., Vanier, M.-T., Bieche, I., Aubourg, P., Crystal, R. G., et al. (2012) · Human Gene Therapy · doi:10.1089/hum.2012.015 · Cited by 91
A Spirulina-Enhanced Diet Provides Neuroprotection in an α-Synuclein Model of Parkinson's Disease
Pabon, M. M., Jernberg, J. N., Morganti, J., Contreras, J., Hudson, C. E., Klein, R. L., & Bickford, P. C. (2012) · PLoS ONE · doi:10.1371/journal.pone.0045256 · Cited by 90
Self-citation
Efficient and stable transduction of dopaminergic neurons in rat substantia nigra by rAAV 2/1, 2/2, 2/5, 2/6.2, 2/7, 2/8 and 2/9
der Perren, A. V., Toelen, J., Carlon, M. S., den Haute, C. V., Coun, F., Heeman, B., Reumers, V., Vandenberghe, L. H., Wilson, J. M., Debyser, Z., et al. (2011) · Gene Therapy · doi:10.1038/gt.2010.179 · Cited by 88
AAV-Mediated Gene Delivery in Adult GM1-Gangliosidosis Mice Corrects Lysosomal Storage in CNS and Improves Survival
Baek, R. C., Broekman, M. L. D., LeRoy, S. G., Tierney, L. A., Sandberg, M. A., d’Azzo, A., Seyfried, T. N., & Sena‐Esteves, M. (2010) · PLoS ONE · doi:10.1371/journal.pone.0013468 · Cited by 90
Obesity, diabetes, and leptin resistance promote tau pathology in a mouse model of disease
Platt, T. L., Beckett, T. L., Köhler, K., Niedowicz, D. M., & Murphy, M. P. (2015) · Neuroscience · doi:10.1016/j.neuroscience.2015.12.011 · Cited by 87
Convection-enhanced delivery and systemic mannitol increase gene product distribution of AAV vectors 5, 8, and 9 and increase gene product in the adult mouse brain
Carty, N., Lee, D. C., Dickey, C. A., Ceballos‐Diaz, C., Jansen‐West, K. R., Golde, T. E., Gordon, M. N., Morgan, D., & Nash, K. R. (2010) · Journal of Neuroscience Methods · doi:10.1016/j.jneumeth.2010.10.010 · Cited by 88
Mimicking Aspects of Frontotemporal Lobar Degeneration and Lou Gehrig's Disease in Rats via TDP-43 Overexpression
Tatom, J. B., Wang, D. B., Dayton, R. D., Skalli, O., Hutton, M. L., Dickson, D. W., & Klein, R. L. (2009) · Molecular Therapy · doi:10.1038/mt.2009.3 · Cited by 78
Self-citation
Current progress and limitations of AAV mediated delivery of protein therapeutic genes and the importance of developing quantitative pharmacokinetic/pharmacodynamic (PK/PD) models
Chowdhury, E. A., Meno‐Tetang, G. M. L., Chang, H. Y., Wu, S., Huang, H. W., Jamier, T., Chandran, J. S., & Shah, D. K. (2021) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2021.01.017 · Cited by 80
Better Targeting, Better Efficiency for Wide-Scale Neuronal Transduction with the Synapsin Promoter and AAV-PHP.B
Jackson, K. L., Dayton, R. D., Deverman, B. E., & Klein, R. L. (2016) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2016.00116 · Cited by 86
Self-citation
Efficient intracerebral delivery of AAV5 vector encoding human ARSA in non-human primate
Colle, M.-A., Piguet, F., Bertrand, L., Raoul, S., Bièche, I., Dubreil, L., Sloothaak, D., Bouquet, C., Moullier, P., Aubourg, P. R., et al. (2009) · Human Molecular Genetics · doi:10.1093/hmg/ddp475 · Cited by 73
Scalable Production of AAV Vectors in Orbitally Shaken HEK293 Cells
Blessing, D., Vachey, G., Pythoud, C., Rey, M. J., Padrun, V., Wurm, F. M., Schneider, B. L., & Déglon, N. (2018) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2018.11.004 · Cited by 74
Optogenetics in the nonhuman primate
Han, X. (2012) · Progress in brain research · doi:10.1016/b978-0-444-59426-6.00011-2 · Cited by 72
Schwann cell targeting via intrasciatic injection of AAV8 as gene therapy strategy for peripheral nerve regeneration
Homs, J., Ariza, L., Pagès, G., Udina, E., Navarro, X., Chillón, M., & Bosch, A. (2011) · Gene Therapy · doi:10.1038/gt.2011.7 · Cited by 67
AAV9-mediated erythropoietin gene delivery into the brain protects nigral dopaminergic neurons in a rat model of Parkinson's disease
Xue, Y.-Q., Ma, B.-F., Zhao, L.-R., Tatom, J. B., Li, B., Jiang, L.-X., Klein, R. L., & Duan, W.-M. (2009) · Gene Therapy · doi:10.1038/gt.2009.113 · Cited by 70
Self-citation
Corrigendum: Better Targeting, Better Efficiency for Wide-Scale Neuronal Transduction with the Synapsin Promoter and AAV-PHP.B
Jackson, K. L., Dayton, R. D., Deverman, B. E., & Klein, R. L. (2016) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2016.00154 · Cited by 68
Self-citation
Adeno-associated viral vector (AAV)-mediated gene transfer in the red nucleus of the adult rat brain: Comparative analysis of the transduction properties of seven AAV serotypes and lentiviral vectors
Blits, B., Derks, S., Twisk, J., Ehlert, E., Prins, J., & Verhaagen, J. (2009) · Journal of Neuroscience Methods · doi:10.1016/j.jneumeth.2009.10.009 · Cited by 66
New tools for investigating astrocyte-to-neuron communication
Li, D., Agulhon, C., Schmidt, E. M., Oheim, M., & Ropert, N. (2013) · Frontiers in Cellular Neuroscience · doi:10.3389/fncel.2013.00193 · Cited by 76
Is Viral Vector Gene Delivery More Effective Using Biomaterials?
Wang, Y., Bruggeman, K. F., Franks, S. J., Gautam, V., Hodgetts, S. I., Harvey, A. R., Williams, R. J., & Nisbet, D. R. (2020) · Advanced Healthcare Materials · doi:10.1002/adhm.202001238 · Cited by 69
CNS-directed gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (Hunter syndrome)
Motas, S., Haurigot, V., Garcia, M., Marcó, S., Ribera, A., Roca, C., Sánchez, X., Sánchez, V., Molas, M., Bertolin, J., et al. (2016) · JCI Insight · doi:10.1172/jci.insight.86696 · Cited by 63
Engineered antibody therapies to counteract mutant huntingtin and related toxic intracellular proteins
Butler, D. C., McLear, J. A., & Messer, A. C. (2011) · Progress in Neurobiology · doi:10.1016/j.pneurobio.2011.11.004 · Cited by 63
Rapid, reproducible transduction of select forebrain regions by targeted recombinant virus injection into the neonatal mouse brain
Pilpel, N., Landeck, N., Klugmann, M., Seeburg, P. H., & Schwarz, M. K. (2009) · Journal of Neuroscience Methods · doi:10.1016/j.jneumeth.2009.05.020 · Cited by 61
Evaluation of the expression pattern of rAAV2/1, 2/5, 2/7, 2/8, and 2/9 serotypes with different promoters in the mouse visual cortex
Scheyltjens, I., Laramée, M., den Haute, C. V., Gijsbers, R., Debyser, Z., Baekelandt, V., Vreysen, S., & Arckens, L. (2015) · The Journal of Comparative Neurology · doi:10.1002/cne.23819 · Cited by 57
Human α-Iduronidase Gene Transfer Mediated by Adeno-Associated Virus Types 1, 2, and 5 in the Brain of Nonhuman Primates: Vector Diffusion and Biodistribution
Ciron, C., Cressant, A., Roux, F., Raoul, S., Chérel, Y., Hantraye, P., Déglon, N., Schwartz, B., Barkats, M., Heard, J., et al. (2009) · Human Gene Therapy · doi:10.1089/hum.2008.155 · Cited by 60
Global gene transfer into the CNS across the BBB after neonatal systemic delivery of single-stranded AAV vectors
Miyake, N., Miyake, K., Yamamoto, M., Hirai, Y., & Shimada, T. (2011) · Brain Research · doi:10.1016/j.brainres.2011.03.014 · Cited by 66
Efficient gene delivery and selective transduction of astrocytes in the mammalian brain using viral vectors
Merienne, N., Douce, J. L., Faivre, E., Déglon, N., & Bonvento, G. (2013) · Frontiers in Cellular Neuroscience · doi:10.3389/fncel.2013.00106 · Cited by 58
Disrupting Function of FK506-Binding Protein 1b/12.6 Induces the Ca2+-Dysregulation Aging Phenotype in Hippocampal Neurons
Gant, J. C., Chen, K.-C., Norris, C. M., Kadish, I., Thibault, O., Blalock, E. M., Porter, N. M., & Landfield, P. W. (2011) · Journal of Neuroscience · doi:10.1523/jneurosci.4805-10.2011 · Cited by 58
Tau expression levels from various adeno‐associated virus vector serotypes produce graded neurodegenerative disease states
Klein, R. L., Dayton, R. D., Tatom, J. B., Diaczynsky, C. G., & Salvatore, M. F. (2008) · European Journal of Neuroscience · doi:10.1111/j.1460-9568.2008.06161.x · Cited by 60
Self-citation
More expansive gene transfer to the rat CNS: AAV PHP.EB vector dose–response and comparison to AAV PHP.B
Dayton, R. D., Grames, M. S., & Klein, R. L. (2018) · Gene Therapy · doi:10.1038/s41434-018-0028-5 · Cited by 55
Self-citation
Intraganglionic AAV6 Results in Efficient and Long-Term Gene Transfer to Peripheral Sensory Nervous System in Adult Rats
Yu, H., Fischer, G. J., Ferhatović, L., Fan, F., Light, A. R., Weihrauch, D., Sapunar, D., Nakai, H., Park, F., & Hogan, Q. H. (2013) · PLoS ONE · doi:10.1371/journal.pone.0061266 · Cited by 50
AAV9 supports wide-scale transduction of the CNS and TDP-43 disease modeling in adult rats
Jackson, K. L., Dayton, R. D., & Klein, R. L. (2015) · Molecular Therapy - Methods & Clinical Development · doi:10.1038/mtm.2015.36 · Cited by 49
Self-citation
Comparison of Adeno-Associated Virus Pseudotype 1, 2, and 8 Vectors Administered by Intramuscular Injection in the Treatment of Murine Phenylketonuria
Rebuffat, A., Harding, C. O., Ding, Z., & Thöny, B. (2009) · Human Gene Therapy · doi:10.1089/hum.2009.127 · Cited by 48
Increased Hippocampal Neurogenesis and Accelerated Response to Antidepressants in Mice with Specific Deletion of CREB in the Hippocampus: Role of cAMP Response-Element Modulator τ
Gundersen, B. B., Briand, L. A., Onksen, J. L., Lay, J. E. L., Kaestner, K. H., & Blendy, J. A. (2013) · Journal of Neuroscience · doi:10.1523/jneurosci.1669-13.2013 · Cited by 51
Astrocytes are key but indirect contributors to the development of the symptomatology and pathophysiology of Huntington's disease
Meunier, C., Mérienne, N., Jollé, C., Déglon, N., & Pellerin, L. (2016) · Glia · doi:10.1002/glia.23022 · Cited by 45
Mouse Gender Influences Brain Transduction by Intravascularly Administered AAV9
Maguire, C. A., Crommentuijn, M. H. W., Mu, D., Hudry, E., Serrano‐Pozo, A., Hyman, B. T., & Tannous, B. A. (2013) · Molecular Therapy · doi:10.1038/mt.2013.95 · Cited by 47
Efficient central nervous system AAVrh10-mediated intrathecal gene transfer in adult and neonate rats
Hordeaux, J., Dubreil, L., Deniaud, J., Iacobelli, F., Moreau, S., Ledevin, M., Guiner, C. L., Blouin, V., Duff, J. L., Mendes-Madeira, A., et al. (2015) · Gene Therapy · doi:10.1038/gt.2014.121 · Cited by 52
Identification of adeno-associated viral vectors suitable for intestinal gene delivery and modulation of experimental colitis
Polyak, S., Mach, A. S., Porvasnik, S., Dixon, L. R., Conlon, T. J., Erger, K. E., Acosta, A. J., Wright, A. J., Campbell‐Thompson, M., Zolotukhin, I., et al. (2011) · American Journal of Physiology-Gastrointestinal and Liver Physiology · doi:10.1152/ajpgi.00562.2010 · Cited by 48
Emerging technologies to study glial cells
Hirbec, H., Déglon, N., Foo, L. C., Goshen, I., Grutzendler, J., Hangen, E., Kreisel, T., Linck, N., Muffat, J., Regio, S., et al. (2020) · Glia · doi:10.1002/glia.23780 · Cited by 51
Transgenic models of Alzheimer's disease: Better utilization of existing models through viral transgenesis
Platt, T. L., Reeves, V. L., & Murphy, M. P. (2013) · Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease · doi:10.1016/j.bbadis.2013.04.017 · Cited by 48
Strategies for targeting primate neural circuits with viral vectors
El-Shamayleh, Y., Ni, A. M., & Horwitz, G. D. (2016) · Journal of Neurophysiology · doi:10.1152/jn.00087.2016 · Cited by 47
Preventing Growth of Brain Tumors by Creating a Zone of Resistance
Maguire, C. A., Meijer, D. H., LeRoy, S. G., Tierney, L. A., Broekman, M. L. D., Costa, F. F., Breakefield, X. O., Stemmer-Rachamimov, A. O., & Sena‐Esteves, M. (2008) · Molecular Therapy · doi:10.1038/mt.2008.168 · Cited by 43
CRTC1 Function During Memory Encoding Is Disrupted in Neurodegeneration
Parra‐Damas, A., Chen, M., Enríquez-Barreto, L., Ortega, L., Acosta, S. P., Camats‐Perna, J., Fullana, M. N., Aguilera, J., Rodrı́guez-Álvarez, J., & Saura, C. A. (2016) · Biological Psychiatry · doi:10.1016/j.biopsych.2016.06.025 · Cited by 49
Adeno-Associated Viral Vectors Serotype 8 for Cell-Specific Delivery of Therapeutic Genes in the Central Nervous System
Pignataro, D., Sucunza, D., Vanrell, L., López‐Franco, E., Dopeso‐Reyes, I. G., Vales, Á., Hommel, M., Rico, A. J., Lanciego, J. L., & González‐Aseguinolaza, G. (2017) · Frontiers in Neuroanatomy · doi:10.3389/fnana.2017.00002 · Cited by 43
GFAP-Driven GFP Expression in Activated Mouse Müller Glial Cells Aligning Retinal Blood Vessels Following Intravitreal Injection of AAV2/6 Vectors
Aartsen, W. M., van Cleef, K. W. R., Pellissier, L. P., Hoek, R. M., Vos, R. M., Blits, B., Ehlert, E. M. E., Balaggan, K. S., Ali, R. R., Verhaagen, J., et al. (2010) · PLoS ONE · doi:10.1371/journal.pone.0012387 · Cited by 41
Intracisternal delivery of AAV9 results in oligodendrocyte and motor neuron transduction in the whole central nervous system of cats
Bucher, T., Dubreil, L., Colle, M.-A., Maquigneau, M., Deniaud, J., Ledevin, M., Moullier, P., & Joussemet, B. (2014) · Gene Therapy · doi:10.1038/gt.2014.16 · Cited by 40
Overexpressing neuroglobin improves functional recovery by inhibiting neuronal apoptosis after spinal cord injury
Lan, W., Lin, J.-H., Chen, X., Wu, C., Zhong, G., Zhang, L., Lin, W., Liu, W.-N., Li, X., & Lin, J.-L. (2014) · Brain Research · doi:10.1016/j.brainres.2014.03.020 · Cited by 38
A simplified purification method for AAV variant by polyethylene glycol aqueous two-phase partitioning
Guo, P., Xiao, X., El‐Gohary, Y., Paredes, J. L., Prasadan, K. L., Shiota, C., Wiersch, J. C., Welsh, C., & Gittes, G. K. (2012) · Bioengineered · doi:10.4161/bioe.22293 · Cited by 38
Adenosine kinase, glutamine synthetase and EAAT2 as gene therapy targets for temporal lobe epilepsy
Young, D., Fong, D. M., Lawlor, P. A., Wu, A. J., Mouravlev, A., McRae, M., Glass, M., Dragunow, M., & During, M. J. (2014) · Gene Therapy · doi:10.1038/gt.2014.82 · Cited by 38
AAV8-mediated overexpression of mPCSK9 in liver differs between male and female mice
Vozenilek, A. E., Blackburn, C. M. R., Schilke, R. M., Chandran, S., Castore, R., Klein, R. L., & Woolard, M. D. (2018) · Atherosclerosis · doi:10.1016/j.atherosclerosis.2018.09.005 · Cited by 36
Self-citation
Adeno-associated virus serotypes 9 and rh10 mediate strong neuronal transduction of the dog brain
Swain, G. P., Prociuk, M. L., Bagel, J. H., O’Donnell, P., Berger, K., Drobatz, K., Gurda, B. L., Haskins, M. E., Sands, M. S., & Vite, C. H. (2013) · Gene Therapy · doi:10.1038/gt.2013.54 · Cited by 34
Adeno‐associated virus‐based Alzheimer's disease mouse models and potential new therapeutic avenues
Ittner, L. M., Klugmann, M., & Ke, Y. D. (2019) · British Journal of Pharmacology · doi:10.1111/bph.14637 · Cited by 36
(Pro)renin receptor knockdown in the paraventricular nucleus of the hypothalamus attenuates hypertension development and AT1 receptor-mediated calcium events
Souza, L. A. C., Worker, C. J., Li, W., Trebak, F., Watkins, T. V., Gayban, A. J. B., Yamasaki, E., Cooper, S. G., Drumm, B. T., & Feng, Y. (2019) · American Journal of Physiology-Heart and Circulatory Physiology · doi:10.1152/ajpheart.00780.2018 · Cited by 34
Heparin-binding correlates with increased efficiency of AAV1- and AAV6-mediated transduction of striated muscle, but negatively impacts CNS transduction
Arnett, A. L. H., Beutler, L. R., Quintana, A., Allen, J. M., Finn, E. E., Palmiter, R. D., & Chamberlain, J. S. (2012) · Gene Therapy · doi:10.1038/gt.2012.60 · Cited by 34
Targeting Homer genes using adeno-associated viral vector: lessons learned from behavioural and neurochemical studies
Klugmann, M., & Szumlinski, K. K. (2008) · Behavioural Pharmacology · doi:10.1097/fbp.0b013e32830c369f · Cited by 34
Intramuscular Delivery of Gene Therapy for Targeting the Nervous System
Tosolini, A. P., & Sleigh, J. N. (2020) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2020.00129 · Cited by 32
A next step in adeno‐associated virus‐mediated gene therapy for neurological diseases: regulation and targeting
Chtarto, A., Bockstael, O., Tshibangu, T., Dewitte, O., Levivier, M., & Tenenbaum, L. (2013) · British Journal of Clinical Pharmacology · doi:10.1111/bcp.12065 · Cited by 33
Absence of Nicotinamide Nucleotide Transhydrogenase in C57BL/6J Mice Exacerbates Experimental Atherosclerosis
Vozenilek, A. E., Vetkoetter, M., Green, J. M., Shen, X., Traylor, J. G., Klein, R. L., Orr, A. W., Woolard, M. D., & Krzywanski, D. M. (2018) · Journal of Vascular Research · doi:10.1159/000486337 · Cited by 31
Self-citation
Lentiviral Vectors: A Powerful Tool to Target Astrocytes In Vivo
Delzor, A., Escartin, C., & Déglon, N. (2013) · Current Drug Targets · doi:10.2174/13894501113146660213 · Cited by 31
Gene Transfer to the CNS Using Recombinant Adeno‐Associated Virus
Stoica, L., Ahmed, S. S., Gao, G., & Sena‐Esteves, M. (2013) · Current Protocols in Microbiology · doi:10.1002/9780471729259.mc14d05s29 · Cited by 29
Optimization of Adeno-Associated Viral Vector-Mediated Gene Delivery to the Hypothalamus
de Backer, M. W. A., Brans, M. A. D., Luijendijk, M. C. M., Garner, K. M., & Adan, R. A. H. (2010) · Human Gene Therapy · doi:10.1089/hum.2009.169 · Cited by 29
Recombinant AAV9-TLK1B Administration Ameliorates Fractionated Radiation-Induced Xerostomia
Shanmugam, P. S. T., Dayton, R. D., Palaniyandi, S., Abreo, F. W., Caldito, G. C., Klein, R. L., & Sunavala‐Dossabhoy, G. (2013) · Human Gene Therapy · doi:10.1089/hum.2012.235 · Cited by 29
Self-citation
Scientific and Regulatory Policy Committee Points to Consider: Nonclinical Research and Development of In Vivo Gene Therapy Products, Emphasizing Adeno-Associated Virus Vectors
Hutt, J. A., Assaf, B. T., Bolon, B. N., Cavagnaro, J. A., Galbreath, E. J., Grubor, B., Kattenhorn, L. M., Romeike, A., & Whiteley, L. O. (2021) · Toxicologic Pathology · doi:10.1177/01926233211041962 · Cited by 27
Adeno-Associated Viral Vector Serotype DJ-Mediated Overexpression of N171-82Q-Mutant Huntingtin in the Striatum of Juvenile Mice Is a New Model for Huntington’s Disease
Jang, M., Lee, S. E., & Cho, I. (2018) · Frontiers in Cellular Neuroscience · doi:10.3389/fncel.2018.00157 · Cited by 25
Gene delivery in tissue engineering and regenerative medicine
Fang, Y. L., Chen, X., & Godbey, W. T. (2014) · Journal of Biomedical Materials Research Part B Applied Biomaterials · doi:10.1002/jbm.b.33354 · Cited by 24

Authors: Klein, R. L., Dayton, R. D., Tatom, J. B., Henderson, K. M., & Henning, P. P.
Affiliations: Louisiana State University Health Sciences Center Shreveport (US)
Corresponding author: Klein, Ronald L
ORCID: Dayton, Robert D
Venue: Molecular Therapy
Publisher: Elsevier BV
Published: 2008-01
Volume: 16
Issue: 1
Pages: 89-96
ISSN: 1525-0016 (ISSN-L)
DOI: 10.1038/sj.mt.6300331
PMC: PMC2987640
OpenAlex: W1992976268
Cited by: 196
References: 29
Citations by year: 2026: 1 · 2025: 2 · 2024: 12 · 2023: 9 · 2022: 5 · 2021: 13 · 2020: 8 · 2019: 8 · 2018: 15 · 2017: 4 · 2016: 13 · 2015: 15 · 2014: 15 · 2013: 20 · 2012: 15
FWCI: 5.18
Type: journal article
Language: en
Open access: hybrid
doi.org
Licence: cc-by-nc-nd
www.elsevier.com/tdm/userlicense/1.0/ (tdm)
creativecommons.org/licenses/by-nc-nd/4.0/ (vor, 3202 d)
Locations: Molecular Therapy (publishedVersion, cc-by-nc-nd)
PubMed (publishedVersion)
PubMed Central (acceptedVersion)
Funders: National Institute of Neurological Disorders and Stroke (R01NS048450)
Topics: Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
Life Sciences › Neuroscience › Neurology › Barrier Structure and Function Studies
Keywords: adeno-associated virus vectors, AAV serotypes, AAV8, AAV9, vector purification, cytomegalovirus promoter, chicken beta-actin promoter, neuronal transduction, hippocampus, GFP expression
MeSH terms: Dependovirus, Animals, Brain Chemistry, Cell Line, Cesium, Chlorides, Genetic Vectors, Hippocampus, Humans, Male, Promoter Regions, Genetic, Serotyping, Transduction, Genetic, Triiodobenzoic Acids, Rats, Sprague-Dawley, Hepatitis B Virus, Woodchuck, Green Fluorescent Proteins, Rats

About these checks

Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.

Statistics and arithmetic are recomputed, not judged.

Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.

What counts as a claim ›How we measure confidence ›How we check this ›

Are you an author, or do you see a problem with this page? Request a review

AAV8, 9, Rh10, Rh43 Vector Gene Transfer in the Rat Brain: Effects of Serotype, Promoter and Purification Method

Abstract

Overview

Integrity signals

Verification

Claims the paper makes

Claims other papers make about this one

Citations

Cites

Cited by

Related

Metadata

Record

About these checks