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, Corinna; Gorbatyuk, Oleg S.; Velardo, Margaret J.; Peden, Carmen S.; Williams, Philip; Zolotukhin, Sergei; Reier, Paul J.; Mandel, Ronald J.; Muzyczka, Nicholas

doi:10.1016/j.ymthe.2004.05.024

Cited by 741Virus-based gene therapy researchRNA Interference and Gene DeliveryBarrier Structure and Function Studies

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

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Burger, Corinna, Gorbatyuk, Oleg S., Velardo, Margaret J., Peden, Carmen S., Williams, Philip, Zolotukhin, Sergei, Reier, Paul J., Mandel, Ronald J. +1 more·2004·Molecular Therapy

DOI 10.1016/j.ymthe.2004.05.024

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Abstract

Recombinant adeno-associated virus 2 (rAAV2) has been shown to deliver genes to neurons effectively in the brain, retina, and spinal cord. The characterization of new AAV serotypes has revealed that they have different patterns of transduction in diverse tissues. We have investigated the tropism and transduction frequency in the central nervous system (CNS) of three different rAAV vector serotypes. The vectors contained AAV2 terminal repeats flanking a green fluorescent protein expression cassette under the control of the synthetic CBA promoter, in AAV1, AAV2, or AAV5 capsids, producing the pseudotypes rAAV2/1, rAAV2/2, and rAAV2/5. Rats were injected with rAAV2/1, rAAV2/2, or rAAV2/5 into selected regions of the CNS, including the hippocampus (HPC), substantia nigra (SN), striatum, globus pallidus, and spinal cord. In all regions injected, the three vectors transduced neurons almost exclusively. All three vectors transduced the SN pars compacta with high efficiency, but rAAV2/1 and rAAV2/5 also transduced the pars reticulata. Moreover, rAAV2/1 showed widespread distribution throughout the entire midbrain. In the HPC, rAAV2/1 and rAAV2/5 targeted the pyramidal cell layers in the CA1-CA3 regions, whereas AAV2/2 primarily transduced the hilar region of the dentate gyrus. In general, rAAV2/1 and rAAV2/5 exhibited higher transduction frequencies than rAAV2/2 in all regions injected, although the differences were marginal in some regions. Retrograde transport of rAAV1 and rAAV5 was also observed in particular CNS areas. These results suggest that vectors based on distinct AAV serotypes can be chosen for specific applications in the nervous system.

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AAV9 can cross the blood-brain barrier and transduce both neurons and astrocytes after intravenous administration.
“AAV9 can cross the blood–brain barrier and transduce both neurons and astrocytes after intravenous administration.7,10–11”
From Characterization of a novel adeno-associated viral vector with preferential oligodendrocyte tropism (2016) · cites this paper as [7]
AAV8 and AAV5 transduce a significantly greater area of the CNS in comparison with AAV2.
“AAV8 and AAV5 transduce a significantly greater area of the CNS in comparison with AAV2”
From Characterization of a novel adeno-associated viral vector with preferential oligodendrocyte tropism (2016) · cites this paper as [7]
No AAV capsid has been described in the literature to exhibit a primary oligodendrocyte tropism when a constitutive promoter drives gene expression.
“No adeno-associated virus (AAV) capsid has been described in the literature to exhibit a primary oligodendrocyte tropism when a constitutive promoter drives gene expression”
From Characterization of a novel adeno-associated viral vector with preferential oligodendrocyte tropism (2016) · cites this paper as [7]
After direct CNS infusion, almost all of the known AAV serotypes exhibit a dominant neuronal tropism.
“after direct CNS infusion, almost all of the known AAV serotypes exhibit a dominant neuronal tropism”
From Characterization of a novel adeno-associated viral vector with preferential oligodendrocyte tropism (2016) · cites this paper as [7]
The vast majority of AAV vectors, such as AAV1, 2, 5, 6, 8 or 9, exhibit a dominant neuronal tropism in the central nervous system.
“The vast majority of AAV vectors, such as AAV1, 2, 5, 6, 8 or 9, exhibit a dominant neuronal tropism in the central nervous system.”
From Characterization of a novel adeno-associated viral vector with preferential oligodendrocyte tropism (2016) · cites this paper as [7]

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Glatzel, M., Flechsig, E., Navarro, B., Klein, M. A., Paterna, J., Büeler, H., & Aguzzi, A. (2000) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.97.1.442 · Cited by 119 · see citing works
Developmental Expression of Platelet-Derived Growth Factor α-Receptor in Neurons and Glial Cells of the Mouse CNS
Nait‐Oumesmar, B., Vignais, L., & Evercooren, A. B. (1997) · Journal of Neuroscience · doi:10.1523/jneurosci.17-01-00125.1997 · Cited by 111 · see citing works
Adeno-associated virus vector-mediated transduction in the cat brain
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Regulation of gene expression in vivo following transduction by two separate rAAV vectors
Rendahl, K. G., Leff, S. E., Otten, G. R., Spratt, S. K., Bohl, D., Roey, M. V., Donahue, B. A., Cohen, L. K., Mandel, R. J., Danos, O., et al. (1998) · Nature Biotechnology · doi:10.1038/nbt0898-757 · Cited by 110 · see citing works
Self-citation
Selective gene expression in brain microglia mediated via adeno-associated virus type 2 and type 5 vectors
Cucchiarini, M., Ren, X., Perides, G., & Terwilliger, E. F. (2003) · Gene Therapy · doi:10.1038/sj.gt.3301925 · Cited by 100 · see citing works
Optic Neuropathy Induced by Reductions in Mitochondrial Superoxide Dismutase
Qi, X., Lewin, A. S., Hauswirth, W. W., & Guy, J. R. (2003) · Investigative Ophthalmology & Visual Science · doi:10.1167/iovs.02-0864 · Cited by 98 · see citing works
Recombinant Adeno-associated Viral Vector-Mediated Glial Cell Line-Derived Neurotrophic Factor Gene Transfer Protects Nigral Dopamine Neurons after Onset of Progressive Degeneration in a Rat Model of Parkinson's Disease
Mandel, R. J., Snyder, R. O., & Leff, S. E. (1999) · Experimental Neurology · doi:10.1006/exnr.1999.7203 · Cited by 93 · see citing works
Self-citation
Gene transfer and expression in oligodendrocytes under the control of myelin basic protein transcriptional control region mediated by adeno-associated virus
Chen, H., McCarty, D., Bruce, A., Suzuki, K., & Suzuki, K. (1998) · Gene Therapy · doi:10.1038/sj.gt.3300547 · Cited by 87 · see citing works
Rate Limiting Steps of AAV Transduction and Implications for Human Gene Therapy
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Recombinant Adeno-associated Virus Serotypes 2– and 5–Mediated Gene Transfer in the Mammalian Brain: Quantitative Analysis of Heparin Co-infusion
Mastakov, M. Y., Baer, K., Kotin, R. M., & During, M. J. (2002) · Molecular Therapy · doi:10.1006/mthe.2002.0564 · Cited by 85 · see citing works
Systemic Hyperosmolality Improves β-Glucuronidase Distribution and Pathology in Murine MPS VII Brain Following Intraventricular Gene Transfer
Ghodsi, A., Stein, C. S., Derksen, T. A., Martins, I., Anderson, R. D., & Davidson, B. L. (1999) · Experimental Neurology · doi:10.1006/exnr.1999.7205 · Cited by 77 · see citing works
Intraneural Colchicine Inhibition of Adenoviral and Adeno-associated Viral Vector Remote Spinal Cord Gene Delivery
Boulis, N. M., Willmarth, N. E., Song, D. K., Feldman, E. L., & Imperiale, M. J. (2003) · Neurosurgery · doi:10.1227/01.neu.0000044459.24519.3e · Cited by 55 · see citing works
Regional localization of dopamine and ionotropic glutamate receptor subtypes in striatolimbic brain regions
Tarazi, F. I., & Baldessarini, R. J. (1999) · Journal of Neuroscience Research · doi:10.1002/(sici)1097-4547(19990215)55:4 · Cited by 51 · see citing works
Tropism of AAV-2 vectors for neurons of the globus pallidus
Tenenbaum, L., Jurysta, F., Stathopoulos, A., et al. (2000) · Neuroreport · doi:10.1097/00001756-200007140-00042 · Cited by 46 · see citing works
Development of hammerhead ribozymes to modulate endogenous gene expression for functional studies
Fritz, J. J., Lewin, A. S., Hauswirth, W. W., Agarwal, A., Grant, M. B., & Shaw, L. C. (2002) · Methods · doi:10.1016/s1046-2023(02)00233-5 · Cited by 24 · see citing works
Textbook of Neurointensive Care
Bell, M. D. D. (2004) · British Journal of Anaesthesia · doi:10.1093/bja/aeh594 · Cited by 22 · see citing works
Uniform Long-Term Gene Expression using Adeno-Associated Virus (AAV) by Ex Vivo Recirculation in Rat-Cardiac Isografts
Asfour, B., Baba, H. A., Scheld, H. H., Hruban, R. H., Hammel, D., & Byrne, B. J. (2002) · The Thoracic and Cardiovascular Surgeon · doi:10.1055/s-2002-35745 · Cited by 18 · see citing works
Gene therapy for disorders of the central nervous system
Aubourg, P. R. (2013) · Handbook of clinical neurology · doi:10.1016/b978-0-444-59565-2.00056-3 · Cited by 8 · see citing works

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Optogenetics in neural systems
Yizhar, O., Fenno, L. E., Davidson, T. J., Mogri, M., & Deisseroth, K. (2011) · Neuron · doi:10.1016/j.neuron.2011.06.004 · Cited by 1,881 · see citing works
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 · see citing works
Adeno-Associated Virus (AAV) as a Vector for Gene Therapy
Naso, M. F., Tomkowicz, B., Perry, W. L., & Strohl, W. R. (2017) · BioDrugs · doi:10.1007/s40259-017-0234-5 · Cited by 1,362 · see citing works
Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: implications for gene therapy using AAV vectors
Boutin, S., Monteilhet, V., Veron, P., Leborgne, C., Benveniste, O., Montus, M. F., & Masurier, C. (2010) · Human Gene Therapy · doi:10.1089/hum.2009.182 · Cited by 970 · see citing works
Functional imaging of hippocampal place cells at cellular resolution during virtual navigation
Dombeck, D. A., Harvey, C. D., Tian, L., Looger, L. L., & Tank, D. W. (2010) · Nature Neuroscience · doi:10.1038/nn.2648 · Cited by 873 · see citing works
Analysis of Transduction Efficiency, Tropism and Axonal Transport of AAV Serotypes 1, 2, 5, 6, 8 and 9 in the Mouse Brain
Aschauer, D. F., Kreuz, S., & Rumpel, S. (2013) · PLoS ONE · doi:10.1371/journal.pone.0076310 · Cited by 522 · see citing works
HDAC3 Is a Critical Negative Regulator of Long-Term Memory Formation
McQuown, S. C., Barrett, R. M., Matheos, D. P., Post, R. J., Rogge, G. A., Alenghat, T., Mullican, S. E., Jones, S., Rusche, J. R., Lazar, M. A., et al. (2011) · The Journal of Neuroscience · doi:10.1523/jneurosci.5052-10.2011 · Cited by 507 · see citing works
An optogenetic toolbox designed for primates
Diester, I., Kaufman, M. T., Mogri, M., Pashaie, R., Goo, W., Yizhar, O., Ramakrishnan, C., Deisseroth, K., & Shenoy, K. V. (2011) · Nature Neuroscience · doi:10.1038/nn.2749 · Cited by 443 · see citing works
Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain
Cearley, C. N., & Wolfe, J. H. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2005.11.015 · Cited by 409 · see citing works
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 · see citing works
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 · see citing works
Gene therapy for lysosomal storage diseases
Sands, M. S., & Davidson, B. L. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2006.01.006 · Cited by 285 · see citing works
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 · 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
Intracellular trafficking of adeno-associated viral vectors
Ding, W., Zhang, L., Yan, Z., & Engelhardt, J. F. (2005) · Gene Therapy · doi:10.1038/sj.gt.3302527 · Cited by 251 · see citing works
Hippocampal focal knockout of CBP affects specific histone modifications, long-term potentiation, and long-term memory
Barrett, R. M., Malvaez, M., Kramar, E., Matheos, D. P., Arrizon, A., Cabrera, S. M., Lynch, G., Greene, R. W., & Wood, M. A. (2011) · Neuropsychopharmacology · doi:10.1038/npp.2011.61 · Cited by 243 · see citing works
Early Oligodendrocyte/Myelin Pathology in Alzheimer's Disease Mice Constitutes a Novel Therapeutic Target
Desai, M. K., Mastrangelo, M. A., Ryan, D. A., Sudol, K. L., Narrow, W. C., & Bowers, W. J. (2010) · The American Journal of Pathology · doi:10.2353/ajpath.2010.100087 · Cited by 232 · see citing works
Chronic Neuron-Specific Tumor Necrosis Factor-Alpha Expression Enhances the Local Inflammatory Environment Ultimately Leading to Neuronal Death in 3xTg-AD Mice
Janelsins, M. C., Mastrangelo, M. A., Park, K. M., Sudol, K. L., Narrow, W. C., Oddo, S., LaFerla, F. M., Callahan, L. M., Federoff, H. J., & Bowers, W. J. (2008) · The American Journal of Pathology · doi:10.2353/ajpath.2008.080528 · Cited by 220 · see citing works
Basic Biology of Adeno-Associated Virus (AAV) Vectors Used in Gene Therapy
Balakrishnan, B., & Jayandharan, G. (2014) · Current Gene Therapy · doi:10.2174/1566523214666140302193709 · Cited by 225 · see citing works
Structurally mapping the diverse phenotype of adeno-associated virus serotype 4
Govindasamy, L., Padron, E., McKenna, R., Muzyczka, N., Kaludov, N., Chiorini, J. A., & Agbandje-McKenna, M. (2006) · Journal of Virology · doi:10.1128/jvi.01536-06 · Cited by 205 · see citing works
Self-citation
Efficient Neuronal Gene Transfer with AAV8 Leads to Neurotoxic Levels of Tau or Green Fluorescent Proteins
Klein, R. L., Dayton, R. D., Leidenheimer, N. J., Jansen, K., Golde, T. E., & Zweig, R. M. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2005.10.008 · Cited by 200 · see citing works
AAV8, 9, Rh10, Rh43 Vector Gene Transfer in the Rat Brain: Effects of Serotype, Promoter and Purification Method
Klein, R. L., Dayton, R. D., Tatom, J. B., Henderson, K. M., & Henning, P. P. (2008) · Molecular Therapy · doi:10.1038/sj.mt.6300331 · Cited by 196 · see citing works
Viral vectors for therapy of neurologic diseases
Choudhury, S. R., Hudry, E., Maguire, C. A., Sena-Esteves, M., Breakefield, X. O., & Grandi, P. (2017) · Neuropharmacology · doi:10.1016/j.neuropharm.2016.02.013 · Cited by 190 · see citing works
rAAV2/7 vector-mediated overexpression of alpha-synuclein in mouse substantia nigra induces protein aggregation and progressive dose-dependent neurodegeneration
Oliveras-Salvá, M., Van der Perren, A., Casadei, N., Stroobants, S., Nuber, S., D’Hooge, R., Van den Haute, C., & Baekelandt, V. (2013) · Molecular Neurodegeneration · doi:10.1186/1750-1326-8-44 · Cited by 191 · see citing works
Viral Strategies for Targeting the Central and Peripheral Nervous Systems
Bedbrook, C. N., Deverman, B. E., & Gradinaru, V. (2018) · Annual Review of Neuroscience · doi:10.1146/annurev-neuro-080317-062048 · Cited by 168 · see citing works
Expanded Repertoire of AAV Vector Serotypes Mediate Unique Patterns of Transduction in Mouse Brain
Cearley, C. N., Vandenberghe, L. H., Parente, M. K., Carnish, E. R., Wilson, J. M., & Wolfe, J. H. (2008) · Molecular Therapy · doi:10.1038/mt.2008.166 · Cited by 182 · see citing works
Targeting photoreceptors via intravitreal delivery using novel, capsid-mutated AAV vectors
Kay, C. N., Ryals, R. C., Aslanidi, G. V., Min, S. H., Ruan, Q., Sun, J., Dyka, F. M., Kasuga, D., Ayala, A. E., Van Vliet, K., et al. (2013) · PLoS ONE · doi:10.1371/journal.pone.0062097 · Cited by 168 · see citing works
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 · see citing works
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 · see citing works
Viral tools for neuroscience
Nectow, A. R., & Nestler, E. J. (2020) · Nature reviews. Neuroscience · doi:10.1038/s41583-020-00382-z · Cited by 159 · see citing works
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 · see citing works
“AAV2 diffuses less readily through both the brain and spinal cord parenchyma when compared against other serotypes, and therefore mediates transduction over a smaller area [19, 28, 32, 33].”
Background
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 · see citing works
Noninvasive and Targeted Gene Delivery into the Brain Using Microbubble-Facilitated Focused Ultrasound
Hsu, P.-H., Wei, K.-C., Huang, C.-Y., Wen, C.-J., Yen, T.-C., Liu, C.-L., Lin, Y.-T., Chen, J.-C., Shen, C.-R., & Liu, H.-L. (2013) · PLoS ONE · doi:10.1371/journal.pone.0057682 · Cited by 136 · see citing works
Characterization of a recombinant adeno-associated virus type 2 Reference Standard Material
Lock, M., McGorray, S., Auricchio, A., Ayuso, E., Beecham, E. J., Blouin-Tavel, V., Bosch, F., Bose, M., Byrne, B. J., Caton, T., et al. (2010) · Human Gene Therapy · doi:10.1089/hum.2009.223 · Cited by 131 · see citing works
Time Course of Transgene Expression After Intrastriatal Pseudotyped rAAV2/1, rAAV2/2, rAAV2/5, and rAAV2/8 Transduction in the Rat
Reimsnider, S., Manfredsson, F. P., Muzyczka, N., & Mandel, R. J. (2007) · Molecular Therapy · doi:10.1038/sj.mt.6300227 · Cited by 125 · see citing works
Self-citation
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 · see citing works
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 · see citing works
Excitatory transmission at thalamo-striatal synapses mediates susceptibility to social stress
Christoffel, D. J., Golden, S. A., Walsh, J. J., Guise, K. G., Heshmati, M., Friedman, A. K., Dey, A., Smith, M., Rebusi, N., Pfau, M., et al. (2015) · Nature Neuroscience · doi:10.1038/nn.4034 · Cited by 101 · see citing works
Structural insights into adeno-associated virus serotype 5
Govindasamy, L., DiMattia, M. A., Gurda, B. L., Halder, S., McKenna, R., Chiorini, J. A., Muzyczka, N., Zolotukhin, S., & Agbandje-McKenna, M. (2013) · Journal of Virology · doi:10.1128/jvi.00867-13 · Cited by 95 · see citing works
Self-citation
Manufacturing and characterization of a recombinant adeno-associated virus type 8 reference standard material
Ayuso, E., Blouin, V., Lock, M., McGorray, S., Leon, X., Alvira, M. R., Auricchio, A., Bucher, S., Chtarto, A., Clark, K. R., et al. (2014) · Human Gene Therapy · doi:10.1089/hum.2014.057 · Cited by 94 · see citing works
Impact of AAV Capsid-Specific T-Cell Responses on Design and Outcome of Clinical Gene Transfer Trials With Recombinant Adeno-Associated Viral Vectors: An Evolving Controversy
Ertl, H. C. J., & High, K. A. (2017) · Human Gene Therapy · doi:10.1089/hum.2016.172 · Cited by 94 · see citing works
AAV9: a potential blood-brain barrier buster
Manfredsson, F. P., Rising, A. C., & Mandel, R. J. (2009) · Molecular Therapy · doi:10.1038/mt.2009.15 · Cited by 94 · see citing works
Self-citation
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 93 · see citing works
Focal Delivery of AAV2/1-transgenes Into the Rat Brain by Localized Ultrasound-induced BBB Opening
Alonso, A., Reinz, E., Leuchs, B., Kleinschmidt, J., Fatar, M., Geers, B., Lentacker, I., Hennerici, M. G., de Smedt, S. C., & Meairs, S. (2013) · Molecular Therapy - Nucleic Acids · doi:10.1038/mtna.2012.64 · Cited by 93 · see citing works
Practical utilization of recombinant AAV vector reference standards: focus on vector genomes titration by free ITR qPCR
D'Costa, S., Blouin, V., Broucque, F., Penaud-Budloo, M., Fçranois, A., Perez, I. C., Le Bec, C., Moullier, P., Snyder, R. O., & Ayuso, E. (2016) · Molecular Therapy - Methods & Clinical Development · doi:10.1038/mtm.2016.19 · Cited by 89 · see citing works
Gene therapy for metachromatic leukodystrophy
Rosenberg, J. B., Kaminsky, S. M., Aubourg, P., Crystal, R. G., & Sondhi, D. (2016) · Journal of Neuroscience Research · doi:10.1002/jnr.23792 · Cited by 91 · see citing works
Transduction of Nonhuman Primate Brain with Adeno-Associated Virus Serotype 1: Vector Trafficking and Immune Response
Hadaczek, P., Forsayeth, J., Mirek, H., Munson, K., Bringas, J., Pivirotto, P., L McBride, J., Davidson, B. L., & Bankiewicz, K. S. (2009) · Human Gene Therapy · doi:10.1089/hum.2008.151 · Cited by 90 · see citing works
Differential Transduction Following Basal Ganglia Administration of Distinct Pseudotyped AAV Capsid Serotypes in Nonhuman Primates
Dodiya, H. B., Bjorklund, T., Stansell III, J., Mandel, R. J., Kirik, D., & Kordower, J. H. (2010) · Molecular Therapy · doi:10.1038/mt.2009.216 · Cited by 86 · see citing works
Self-citation
Persistent repression of tau in the brain using engineered zinc finger protein transcription factors
Wegmann, S., DeVos, S. L., Zeitler, B., Marlen, K., Bennett, R. E., Perez-Rando, M., MacKenzie, D., Yu, Q., Commins, C., Bannon, R. N., et al. (2021) · Science Advances · doi:10.1126/sciadv.abe1611 · Cited by 77 · see citing works
A novel role for brain interleukin-6: facilitation of cognitive flexibility in rat orbitofrontal cortex
Donegan, J. J., Girotti, M., Weinberg, M. S., & Morilak, D. A. (2014) · The Journal of Neuroscience · doi:10.1523/jneurosci.3968-13.2014 · Cited by 77 · see citing works
Normalization of Dyrk1A expression by AAV2/1-shDyrk1A attenuates hippocampal-dependent defects in the Ts65Dn mouse model of down syndrome
Altafaj, X., Martín, E. D., Ortiz-Abalia, J., Valderrama, A., Lao-Peregrín, C., Dierssen, M., & Fillat, C. (2013) · Neurobiology of Disease · doi:10.1016/j.nbd.2012.11.017 · Cited by 75 · see citing works
Adeno‐Associated Viral Vectors for Anterograde Axonal Tracing with Fluorescent Proteins in Nontransgenic and Cre Driver Mice
Harris, J. A., Wook Oh, S., & Zeng, H. (2012) · Current Protocols in Neuroscience · doi:10.1002/0471142301.ns0120s59 · Cited by 72 · see citing works
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 · see citing works
Examining the cross-reactivity and neutralization mechanisms of a panel of mAbs against adeno-associated virus serotypes 1 and 5
Harbison, C. E., Weichert, W. S., Gurda, B. L., Chiorini, J. A., Agbandje-McKenna, M., & Parrish, C. R. (2012) · Journal of General Virology · doi:10.1099/vir.0.035113-0 · Cited by 54 · see citing works
Accurate Titration of Infectious AAV Particles Requires Measurement of Biologically Active Vector Genomes and Suitable Controls
François, A., Bouzelha, M., Lecomte, E., Broucque, F., Penaud-Budloo, M., Adjali, O., Moullier, P., Blouin, V., & Ayuso, E. (2018) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2018.07.004 · Cited by 50 · see citing works
Designing and optimizing AAV-mediated gene therapy for neurodegenerative diseases: from bench to bedside
Xu, L., Yao, S., Ding, Y. E., Xie, M., Feng, D., Sha, P., Tan, L., Bei, F., & Yao, Y. (2024) · Journal of Translational Medicine · doi:10.1186/s12967-024-05661-2 · Cited by 48 · see citing works
Enhanced Gene Transfer Efficiency in the Murine Striatum and an Orthotopic Glioblastoma Tumor Model, Using AAV-7- and AAV-8-Pseudotyped Vectors
Harding, T. C., Dickinson, P. J., Roberts, B. N., Yendluri, S., Gonzalez-Edick, M., Lecouteur, R. A., & Jooss, K. U. (2006) · Human Gene Therapy · doi:10.1089/hum.2006.17.807 · Cited by 45 · see citing works
Striatal readministration of rAAV vectors reveals an immune response against AAV2 capsids that can be circumvented
Peden, C. S., Manfredsson, F. P., Reimsnider, S. K., Poirier, A. E., Burger, C., Muzyczka, N., & Mandel, R. J. (2009) · Molecular Therapy · doi:10.1038/mt.2008.284 · Cited by 44 · see citing works
Self-citation
Systematic comparison of adeno‐associated virus and biotinylated dextran amine reveals equivalent sensitivity between tracers and novel projection targets in the mouse brain
Wang, Q., Henry, A. M., Harris, J. A., Oh, S. W., Joines, K. M., Nyhus, J., Hirokawa, K. E., Dee, N., Mortrud, M., Parry, S., et al. (2014) · Journal of Comparative Neurology · doi:10.1002/cne.23567 · Cited by 44 · see citing works
Regulatory T Cells Control Uveoretinitis Induced by Pathogenic Th1 Cells Reacting to a Specific Retinal Neoantigen
Terrada, C., Fisson, S., De Kozak, Y., Kaddouri, M., Lehoang, P., Klatzmann, D., Salomon, B. L., & Bodaghi, B. (2006) · The Journal of Immunology · doi:10.4049/jimmunol.176.12.7171 · Cited by 38 · see citing works
Rationally designed AAV2 and AAVrh8R capsids provide improved transduction in the retina and brain
Sullivan, J. A., Stanek, L. M., Lukason, M. J., Bu, J., Osmond, S. R., Barry, E. A., O’Riordan, C. R., Shihabuddin, L. S., Cheng, S. H., & Scaria, A. (2018) · Gene Therapy · doi:10.1038/s41434-018-0017-8 · Cited by 37 · see citing works
Adeno-Associated Virus Expression of α-Synuclein as a Tool to Model Parkinson’s Disease: Current Understanding and Knowledge Gaps
Huntington, T. E., & Srinivasan, R. (2021) · Aging and disease · doi:10.14336/ad.2021.0517 · Cited by 34 · see citing works
Distinct roles for the deacetylase domain of HDAC3 in the hippocampus and medial prefrontal cortex in the formation and extinction of memory
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Quantification of Adeno-Associated Virus with Safe Nucleic Acid Dyes
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Authors: Burger, C., Gorbatyuk, O. S., Velardo, M. J., Peden, C. S., Williams, P., Zolotukhin, S., Reier, P. J., Mandel, R. J., & Muzyczka, N.
Affiliations: University of Florida (US)
Corresponding author: Muzyczka, Nicholas
ORCID: Gorbatyuk, Oleg S.
Williams, Philip
Mandel, Ronald J.
Venue: Molecular Therapy
Publisher: Elsevier BV
Published: 2004-08
Volume: 10
Issue: 2
Pages: 302-317
ISSN: 1525-0016 (ISSN-L)
DOI: 10.1016/j.ymthe.2004.05.024
OpenAlex: W2003456064
Cited by: 741
References: 85
Citations by year: 2026: 7 · 2025: 27 · 2024: 15 · 2023: 17 · 2022: 25 · 2021: 38 · 2020: 26 · 2019: 38 · 2018: 33 · 2017: 35 · 2016: 24 · 2015: 41 · 2014: 42 · 2013: 47 · 2012: 35
FWCI: 20.82
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, 4450 d)
Locations: Molecular Therapy (publishedVersion, cc-by-nc-nd)
PubMed (publishedVersion)
Funders: National Institutes of Health
National Institute of Neurological Disorders and Stroke (NS P0136302)
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: recombinant adeno-associated virus vectors, AAV serotypes, AAV1, AAV2, AAV5, cell tropism, neuronal transduction, central nervous system, substantia nigra, hippocampus, striatum, globus pallidus, spinal cord, retrograde transport
MeSH terms: Dependovirus, Animals, Capsid, Central Nervous System, Genetic Vectors, Male, Neurons, Tissue Distribution, Transduction, Genetic, Genetic Therapy, Rats, Sprague-Dawley, Green Fluorescent Proteins, Rats

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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

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