Comparative Analysis of Adeno-Associated Viral Vector Serotypes 1, 2, 5, 7, And 8 in Mouse Brain
Abstract
Recombinant adeno-associated virus serotype 2 (rAAV2) vectors have been shown to deliver genes effectively to neurons in the brain, retina, and spinal cord. The characterization of new AAV serotypes revealed different patterns of transduction in a diverse array of tissues (Gao, G., Vandenberghe, L.H., and Wilson, J.M. [2005]. Curr. Gene Ther. 5, 285-297). Here, we extensively compare the neural tropism of human-derived rAAVs (types 2/1, 2, and 2/5) with nonhuman primate-derived rAAVs (types 2/7 and 2/8) in adult mouse brain. Mice were injected with rAAV type 2/1, 2, 2/5, 2/7, or 2/8 via the caudate-putamen and substantia nigra. Intrahippocampal injections were also performed for rAAV2/7 and rAAV2/8. In all regions injected, the vectors transduced neurons almost exclusively. Retrograde transduction of all rAAV pseudotypes was also observed in particular CNS areas. At high titers, all rAAV pseudotypes transduced comparable brain volumes in all targeted regions except for rAAV2, which transduced much smaller brain volumes. A dose-range comparison of intrastriatally injected rAAV types 2/5, 2/7, and 2/8 highlighted that the transduction efficiency, as determined by transduced volume and biophotonic imaging of green fluorescent protein expression intensity, was significantly higher for rAAV2/5 and rAAV2/7 compared with rAAV2/8 at low titers, whereas all three serotypes performed equally well at higher doses. These results demonstrate the use and efficiency of both human- and nonhuman primate-derived rAAV vectors for disease modeling and their potential for gene therapy.
Integrity signals
Registry
Cites
Statistics
Claims the paper makes
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?
AAV8 and AAV9 exhibit a dominant neuronal tropism after direct injection into the rodent striatum as well as in other brain structures.
“AAV8 and AAV9 exhibit a dominant neuronal tropism after direct injection into the rodent striatum9,13,16,27 as well as in other brain structures.12,28,29”
From Characterization of a novel adeno-associated viral vector with preferential oligodendrocyte tropism (2016) · cites this paper as [28]
Cites
The Mouse Brain in Stereotaxic Coordinates
Paxinos, G. (2001) · Cited by 12,859 · see citing works
A Third-Generation Lentivirus Vector with a Conditional Packaging System
Dull, T., Zufferey, R., Kelly, M., Mandel, R. J., Nguyen, M., Trono, D., & Naldini, L. (1998) · Journal of Virology · doi:10.1128/jvi.72.11.8463-8471.1998 · Cited by 3,341 · see citing works
Manno, C. S., Pierce, G. F., Arruda, V. R., Glader, B., Ragni, M., Rasko, J. J. E., Ozelo, M. C., Hoots, K., Blatt, P., Konkle, B., et al. (2006) · Nature Medicine · doi:10.1038/nm1358 · Cited by 2,163 · see citing works
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 · see citing works
Self-citationAnalysis 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
Xiao, X., Li, J., & Samulski, R. J. (1998) · Journal of Virology · doi:10.1128/jvi.72.3.2224-2232.1998 · Cited by 1,381 · see citing works
Clades of Adeno-Associated Viruses Are Widely Disseminated in Human Tissues
Gao, G., Vandenberghe, L. H., Alvira, M. R., Lu, Y., Calcedo, R., Zhou, X., & Wilson, J. M. (2004) · Journal of Virology · doi:10.1128/jvi.78.12.6381-6388.2004 · Cited by 1,070 · see citing works
Self-citationBurger, 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
Davidson, B. L., Stein, C. S., Heth, J., 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.050581197 · Cited by 679 · see citing works
Design-based stereology in neuroscience
Schmitz, C., & Hof, P. R. (2004) · Neuroscience · doi:10.1016/j.neuroscience.2004.08.050 · Cited by 676 · see citing works
Circuit-breakers: optical technologies for probing neural signals and systems
Zhang, F., Aravanis, A. M., Adamantidis, A., de Lecea, L., & Deisseroth, K. A. (2007) · Nature reviews. Neuroscience · doi:10.1038/nrn2192 · Cited by 623 · see citing works
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 · see citing works
New recombinant serotypes of AAV vectors
Gao, G., Vandenberghe, L., & Wilson, J. (2005) · Current Gene Therapy · doi:10.2174/1566523054065057 · Cited by 487 · see citing works
Self-citationGrieger, J. C., & Samulski, R. J. (2005) · Journal of Virology · doi:10.1128/jvi.79.15.9933-9944.2005 · Cited by 419 · see citing works
Cearley, C. N., & Wolfe, J. H. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2005.11.015 · Cited by 409 · see citing works
Auricchio, A., Hildinger, M., O'Connor, E., Gao, G., & Wilson, J. M. (2001) · Human Gene Therapy · doi:10.1089/104303401450988 · Cited by 296 · see citing works
Self-citationPeden, C. S., Burger, C., Muzyczka, N., & Mandel, R. J. (2004) · Journal of Virology · doi:10.1128/jvi.78.12.6344-6359.2004 · Cited by 222 · see citing works
Recombinant Adeno-associated Virus Transduction and Integration
Schultz, B. R., & Chamberlain, J. S. (2008) · Molecular Therapy · doi:10.1038/mt.2008.103 · Cited by 216 · see citing works
Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid
Vandenberghe, L. H., Wang, L., Somanathan, S., Zhi, Y., Figueredo, J., Calcedo, R., Sanmiguel, J., Desai, R. A., Chen, C. S., Johnston, J., et al. (2006) · Nature Medicine · doi:10.1038/nm1445 · Cited by 208 · see citing works
Self-citationRecombinant 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
Hybrid Vectors Based on Adeno-Associated Virus Serotypes 2 and 5 for Muscle-Directed Gene Transfer
Hildinger, M., Auricchio, A., Gao, G., Wang, L., Chirmule, N. B., & Wilson, J. M. (2001) · Journal of Virology · doi:10.1128/jvi.75.13.6199-6203.2001 · Cited by 204 · see citing works
Self-citationBroekman, M. L. D., Comer, L. A., Hyman, B. T., & Sena‐Esteves, M. (2006) · Neuroscience · doi:10.1016/j.neuroscience.2005.11.057 · Cited by 201 · see citing works
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
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
Adeno-Associated Virus-Mediated Gene Transfer to the Brain: Duration and Modulation of Expression
Lo, W. D., Qu, G., Sferra, T. J., Clark, R., Chen, R., & Johnson, P. R. (1999) · Human Gene Therapy · doi:10.1089/10430349950018995 · Cited by 188 · 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
Self-citationFranich, N. R., Fitzsimons, H. L., Fong, D. M., Klugmann, M., During, M. J., & Young, D. (2008) · Molecular Therapy · doi:10.1038/mt.2008.50 · Cited by 157 · see citing works
Baekelandt, V, Eggermont, K., Michiels, M., Nuttin, B., & Debyser, Z. (2003) · Gene Therapy · doi:10.1038/sj.gt.3302094 · Cited by 153 · see citing works
Self-citationHoward, D. B., Powers, K., Wang, Y., & Harvey, B. K. (2008) · Virology · doi:10.1016/j.virol.2007.10.007 · Cited by 149 · see citing works
Hellström, M., Ruitenberg, M. J., Pollett, M. A., Ehlert, E. M. E., Twisk, J., Verhaagen, J., & Harvey, A. R. (2009) · Gene Therapy · doi:10.1038/gt.2008.178 · Cited by 142 · see citing works
Characterization of Lentiviral Vector-Mediated Gene Transfer in Adult Mouse Brain
Baekelandt, V., Claeys, A., Eggermont, K., Lauwers, E., Strooper, B. D., Nuttin, B., & Debyser, Z. (2002) · Human Gene Therapy · doi:10.1089/10430340252899019 · Cited by 137 · see citing works
Self-citationComparison of Adenoviral and Adeno-Associated Viral Vectors for Pancreatic Gene Delivery In Vivo
Wang, A. Y., Peng, P. D., Ehrhardt, A., Storm, T. A., & Kay, M. A. (2004) · Human Gene Therapy · doi:10.1089/104303404322959551 · Cited by 128 · see citing works
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
Efficient Retrograde Neuronal Transduction Utilizing Self-complementary AAV1
Hollis, E. R., II, Kadoya, K., Hirsch, M. L., Samulski, R. J., & Tuszynski, M. H. (2008) · Molecular Therapy · doi:10.1038/sj.mt.6300367 · Cited by 124 · see citing works
Immunological Aspects of Recombinant Adeno-Associated Virus Delivery to the Mammalian Brain
Mastakov, M. Y., Baer, K., Symes, C. W., Leichtlein, C. B., Kotin, R. M., & During, M. J. (2002) · Journal of Virology · doi:10.1128/jvi.76.16.8446-8454.2002 · Cited by 114 · see citing works
Recombinant Adeno-Associated Viral Vectors in the Nervous System
Burger, C., Nash, K., & Mandel, R. J. (2005) · Human Gene Therapy · doi:10.1089/hum.2005.16.781 · Cited by 110 · see citing works
Paterna, J., Feldon, J., & Büeler, H. (2004) · Journal of Virology · doi:10.1128/jvi.78.13.6808-6817.2004 · Cited by 96 · see citing works
Dodge, J. C., Clarke, J. L., Song, A., Bu, J., Yang, W., Taksir, T. V., Griffiths, D. A., Zhao, M. A., Schuchman, E. H., Cheng, S. H., et al. (2005) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0509062102 · Cited by 90 · see citing works
Potential of AAV vectors in the treatment of metabolic disease
Alexander, I. E., Cunningham, S. C., Logan, G. J., & Christodoulou, J. (2008) · Gene Therapy · doi:10.1038/gt.2008.64 · Cited by 88 · see citing works
Franz, C. K., Federici, T., Yang, J., Backus, C., Oh, S. S., Teng, Q., Carlton, E. F., Bishop, K. M., Gasmi, M., Bartus, R. T., et al. (2008) · Neurobiology of Disease · doi:10.1016/j.nbd.2008.12.003 · Cited by 77 · see citing works
Verity, M. A. (1995) · Elsevier eBooks · doi:10.1016/b978-0-12-294375-1.50012-x · Cited by 69 · see citing works
Survival advantage of neonatal CNS gene transfer for late infantile neuronal ceroid lipofuscinosis
Sondhi, D., Peterson, D. A., Edelstein, A., del Fierro, K., Hackett, N. R., & Crystal, R. G. (2008) · Experimental Neurology · doi:10.1016/j.expneurol.2008.04.022 · Cited by 68 · see citing works
Cunningham, J., Pivirotto, P., Bringas, J., Suzuki, B. M., Vijay, S., Sanftner, L. M., Kitamura, M., Chan, C. M., & Bankiewicz, K. S. (2008) · Molecular Therapy · doi:10.1038/mt.2008.111 · Cited by 68 · see citing works
Takeda, S., Takahashi, M., Mizukami, H., Kobayashi, E., Takeuchi, K., Hakamata, Y., Kaneko, T., Yamamoto, H., Ito, C., Ozawa, K., et al. (2004) · Nephron Experimental Nephrology · doi:10.1159/000077378 · Cited by 63 · see citing works
Riban, V., Fitzsimons, H. L., & During, M. J. (2008) · Epilepsia · doi:10.1111/j.1528-1167.2008.01743.x · Cited by 53 · see citing works
Watson, D. J., Passini, M. A., & Wolfe, J. H. N. (2005) · Human Gene Therapy · doi:10.1089/hum.2005.16.49 · Cited by 45 · see citing works
Mandel, R. J., Bürger, C., & Snyder, R. O. (2007) · Experimental Neurology · doi:10.1016/j.expneurol.2007.08.008 · Cited by 44 · see citing works
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 · see citing works
Adeno‐associated virus‐mediated gene delivery approaches for the treatment of CNS disorders
Terzi, D., & Zachariou, V. (2008) · Biotechnology Journal · doi:10.1002/biot.200800284 · Cited by 42 · see citing works
Gene therapeutic strategies for neurodegenerative diseases.
Baekelandt, V., Strooper, B. D., Nuttin, B., & Debyser, Z. (2000) · PubMed · Cited by 40 · see citing works
Self-citationGene Delivery to the Mouse Brain with Adeno-Associated Virus
Passini, M. A., Watson, D. J., & Wolfe, J. H. N. (2004) · Humana Press eBooks · doi:10.1385/1-59259-650-9:225 · Cited by 32 · see citing works
Asokan, A., Johnson, J. S., Li, C., & Samulski, R. J. (2008) · Gene Therapy · doi:10.1038/gt.2008.127 · Cited by 28 · see citing works
Elsworth, J. D., Redmond, D. E., Léránth, C., Bjugstad, K. B., Sladek, J. R., Collier, T. J., Foti, S., Samulski, R. J., Vives, K. P., & Roth, R. H. (2008) · Experimental Neurology · doi:10.1016/j.expneurol.2008.01.026 · Cited by 27 · see citing works
Bockstael, O., Chtarto, A., Wakkinen, J., Yang, X. L., Melas, C., Levivier, M., Brotchi, J., & Tenenbaum, L. (2008) · Human Gene Therapy · doi:10.1089/hum.2008.099 · Cited by 20 · see citing works
Denovan‐Wright, E. M., Attis, M., Rodríguez-Lebrón, E., & Mandel, R. J. (2008) · Journal of Neuroscience Research · doi:10.1002/jnr.21636 · Cited by 17 · see citing works
Belur, L. R., Kaemmerer, W. F., McIvor, R. S., & Low, W. C. (2008) · Archives of Virology · doi:10.1007/s00705-008-0222-7 · Cited by 10 · see citing works
Immune-Directed Gene Therapeutic Development for Alzheimer’s, Prion, and Parkinson’s Diseases
Maguire‐Zeiss, K. A., & Federoff, H. J. (2008) · Journal of Neuroimmune Pharmacology · doi:10.1007/s11481-008-9133-3 · Cited by 9 · see citing works
Benedetto, B. D., Wefers, B., Wurst, W., & Kühn, R. (2008) · Molecular Biotechnology · doi:10.1007/s12033-008-9125-9 · Cited by 5 · see citing works
Safety of Adeno-Associated Viral Vectors
Lin, S., & Ertl, H. C. J. (2008) · Future Virology · doi:10.2217/17460794.3.5.491 · Cited by 3 · see citing works
Identification of Novel Adeno-Associated Virus Serotypes for Use as Vectors
Vandenberghe, L. H., Wilson, J. M., & Gao, G. (2006) · Elsevier eBooks · doi:10.1016/b978-012397632-1/50003-4 · Cited by 1 · see citing works
Self-citation
Flagged first, then by citations
Cited by
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 · see citing works
A Designer AAV Variant Permits Efficient Retrograde Access to Projection Neurons
Tervo, D. G. R., Hwang, B., Viswanathan, S., Gaj, T., Lavzin, M., Ritola, K., Lindo, S. E., Michael, S. C., Кулешова, Е. П., Ojala, D. S., et al. (2016) · Neuron · doi:10.1016/j.neuron.2016.09.021 · Cited by 1,170 · see citing works
Functional identification of an aggression locus in the mouse hypothalamus
Lin, D., Boyle, M. P., Dollar, P., Lee, H., Lein, E. S., Perona, P., & Anderson, D. J. (2011) · Nature · doi:10.1038/nature09736 · Cited by 914 · see citing works
Circuit-breakers: optical technologies for probing neural signals and systems
Zhang, F., Aravanis, A. M., Adamantidis, A., de Lecea, L., & Deisseroth, K. A. (2007) · Nature reviews. Neuroscience · doi:10.1038/nrn2192 · Cited by 623 · see citing works
Cardin, J. A., Carlén, M., Meletis, K., Knoblich, U., Zhang, F., Deisseroth, K. A., Tsai, L., & Moore, C. I. (2010) · Nature Protocols · doi:10.1038/nprot.2009.228 · Cited by 542 · see citing works
Cortical representations of olfactory input by trans-synaptic tracing
Miyamichi, K., Amat, F., Moussavi, F., Wang, C., Wickersham, I. R., Wall, N. R., Taniguchi, H., Tasic, B., Huang, Z. J., He, Z., et al. (2010) · Nature · doi:10.1038/nature09714 · Cited by 546 · see citing works
Aschauer, D. F., Kreuz, S., & Rumpel, S. (2013) · PLoS ONE · doi:10.1371/journal.pone.0076310 · Cited by 522 · see citing works
Modeling Parkinson’s Disease With the Alpha-Synuclein Protein
Gómez-Benito, M., Granado, N., García‐Sanz, P., Michel, A., Dumoulin, M., & Moratalla, R. (2020) · Frontiers in Pharmacology · doi:10.3389/fphar.2020.00356 · Cited by 445 · see citing works
Long‐range connectivity of mouse primary somatosensory barrel cortex
Aronoff, R., Matyas, F., Mateo, C., Ciron, C., Schneider, B., & Petersen, C. C. H. (2010) · European Journal of Neuroscience · doi:10.1111/j.1460-9568.2010.07264.x · Cited by 323 · see citing works
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 319 · 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
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 · see citing works
Applications of CRISPR–Cas systems in neuroscience
Heidenreich, M., & Zhang, F. (2015) · Nature reviews. Neuroscience · doi:10.1038/nrn.2015.2 · Cited by 282 · see citing works
Viral vectors for gene delivery to the central nervous system
Lentz, T. B., Gray, S. J., & Samulski, R. J. (2011) · Neurobiology of Disease · doi:10.1016/j.nbd.2011.09.014 · Cited by 274 · see citing works
Intracellular transport of recombinant adeno-associated virus vectors
Nonnenmacher, M., & Weber, T. (2012) · Gene Therapy · doi:10.1038/gt.2012.6 · Cited by 258 · see citing works
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 · see citing works
Recombinant Adeno-associated Virus Transduction and Integration
Schultz, B. R., & Chamberlain, J. S. (2008) · Molecular Therapy · doi:10.1038/mt.2008.103 · Cited by 216 · see citing works
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 207 · see citing works
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
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
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
Self-citationComparison of AAV Serotypes for Gene Delivery to Dorsal Root Ganglion Neurons
Mason, M. R. J., Ehlert, E., Eggers, R., Pool, C. W., Hermening, S., Huseinovic, A., Timmermans, E., Blits, B., & Verhaagen, J. (2010) · Molecular Therapy · doi:10.1038/mt.2010.19 · Cited by 183 · 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
Self-citationMolecular 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 · see citing works
Ip, C. W., Klaus, L.-C., Karikari, A. A., Visanji, N. P., Brotchie, J. M., Lang, A. E., Volkmann, J., & Koprich, J. B. (2017) · Acta Neuropathologica Communications · doi:10.1186/s40478-017-0416-x · Cited by 173 · see citing works
Automated quantitative gait analysis in animal models of movement disorders
Vandeputte, C., Taymans, J., Casteels, C., Coun, F., Ni, Y., Laere, K. V., & Baekelandt, V. (2010) · BMC Neuroscience · doi:10.1186/1471-2202-11-92 · Cited by 169 · see citing works
Self-citationViral 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
Rapid evolution of blood-brain-barrier-penetrating AAV capsids by RNA-driven biopanning
Nonnenmacher, M., Wang, W., Child, M. A., Ren, X.-Q., Huang, C., Ren, A. Z., Tocci, J., Chen, Q., Bittner, K., Tyson, K., et al. (2021) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2020.12.006 · Cited by 168 · see citing works
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
Lipid-mediated delivery of RNA is more efficient than delivery of DNA in non-dividing cells
Zou, S., Scarfo, K. A., Nantz, M. H., & Hecker, J. G. (2010) · International Journal of Pharmaceutics · doi:10.1016/j.ijpharm.2010.01.019 · Cited by 161 · see citing works
Franich, N. R., Fitzsimons, H. L., Fong, D. M., Klugmann, M., During, M. J., & Young, D. (2008) · Molecular Therapy · doi:10.1038/mt.2008.50 · Cited by 157 · see citing works
Nassi, J. J., Cepko, C. L., Born, R. T., & Beier, K. T. (2015) · Frontiers in Neuroanatomy · doi:10.3389/fnana.2015.00080 · Cited by 156 · see citing works
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 156 · see citing works
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 154 · 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
Self-citationLights, fiber, action! A primer on in vivo fiber photometry
Simpson, E. H., Akam, T. E., Patriarchi, T., Blanco-Pozo, M., Burgeno, L. M., Mohebi, A., Cragg, S. J., & Walton, M. E. (2023) · Neuron · doi:10.1016/j.neuron.2023.11.016 · Cited by 150 · see citing works
Howard, D. B., Powers, K., Wang, Y., & Harvey, B. K. (2008) · Virology · doi:10.1016/j.virol.2007.10.007 · Cited by 149 · see citing works
Recent progress and considerations for AAV gene therapies targeting the central nervous system
Lykken, E. A., Shyng, C., Edwards, R. J., Rozenberg, A. J., & Gray, S. J. (2018) · Journal of Neurodevelopmental Disorders · doi:10.1186/s11689-018-9234-0 · Cited by 140 · see citing works
Metachromatic Leukodystrophy: Diagnosis, Modeling, and Treatment Approaches
Shaimardanova, A. A., Chulpanova, D. S., Solovyeva, V. V., Mullagulova, A. I., Kitaeva, K. V., Allegrucci, C., & Rizvanov, A. A. (2020) · Frontiers in Medicine · doi:10.3389/fmed.2020.576221 · Cited by 143 · see citing works
Hellström, M., Ruitenberg, M. J., Pollett, M. A., Ehlert, E. M. E., Twisk, J., Verhaagen, J., & Harvey, A. R. (2009) · Gene Therapy · doi:10.1038/gt.2008.178 · Cited by 142 · see citing works
Adeno-Associated Viral Vectors and Their Redirection to Cell-Type Specific Receptors
Michelfelder, S., & Trepel, M. (2009) · Advances in genetics · doi:10.1016/s0065-2660(09)67002-4 · Cited by 134 · see citing works
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 · see citing works
McFarland, N. R., Fan, Z., Xu, K., Schwarzschild, M. A., Feany, M. Β., Hyman, B. T., & McLean, P. J. (2009) · Journal of Neuropathology & Experimental Neurology · doi:10.1097/nen.0b013e3181a24b53 · Cited by 131 · see citing works
Markakis, E. A., Vives, K. P., Bober, J., Leichtle, S. W., Léránth, C., Beecham, J., Elsworth, J. D., Roth, R. H., Samulski, R. J., & Redmond, D. E. (2009) · Molecular Therapy · doi:10.1038/mt.2009.286 · Cited by 130 · see citing works
Seung Taik, L. I. M., Airavaara, M., & Harvey, B. K. (2009) · Pharmacological Research · doi:10.1016/j.phrs.2009.10.002 · Cited by 126 · see citing works
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
Efficient Retrograde Neuronal Transduction Utilizing Self-complementary AAV1
Hollis, E. R., II, Kadoya, K., Hirsch, M. L., Samulski, R. J., & Tuszynski, M. H. (2008) · Molecular Therapy · doi:10.1038/sj.mt.6300367 · Cited by 124 · see citing works
Adeno-Associated Viral Vectors in Neuroscience Research
Haggerty, D. L., Grecco, G. G., Reeves, K. C., & Atwood, B. K. (2019) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2019.11.012 · Cited by 123 · see citing works
Rothermel, M., Brunert, D., Zabawa, C., Díaz-Quesada, M., & Wachowiak, M. (2013) · Journal of Neuroscience · doi:10.1523/jneurosci.1618-13.2013 · Cited by 116 · see citing works
A glance at genome editing with CRISPR–Cas9 technology
Barman, A., Deb, B., & Chakraborty, S. (2019) · Current Genetics · doi:10.1007/s00294-019-01040-3 · Cited by 105 · see citing works
der Perren, A. V., Toelen, J., Casteels, C., Macchi, F., Rompuy, A. V., Sarre, S., Casadei, N., Nuber, S., Himmelreich, U., Garcia, M. I. O., et al. (2015) · Neurobiology of Aging · doi:10.1016/j.neurobiolaging.2014.11.015 · Cited by 95 · see citing works
Self-citationAdeno-Associated Viral Vectors for Mapping, Monitoring, and Manipulating Neural Circuits
Betley, J. N., & Sternson, S. M. (2011) · Human Gene Therapy · doi:10.1089/hum.2010.204 · Cited by 102 · see citing works
Berton, O., Guigoni, C., Li, Q., Bioulac, B. H., Aubert, I., Gross, C. E., Dileone, R., Nestler, E. J., & Bézard, E. (2009) · Biological Psychiatry · doi:10.1016/j.biopsych.2009.04.005 · Cited by 98 · see citing works
Gérard, M., Deleersnijder, A., Daniëls, V., Schreurs, S., Munck, S., Reumers, V., Pottel, H., Engelborghs, Y., den Haute, C. V., Taymans, J., et al. (2010) · Journal of Neuroscience · doi:10.1523/jneurosci.5983-09.2010 · Cited by 96 · see citing works
Self-citationGene Therapy for the Nervous System: Challenges and New Strategies
Maguire, C. A., Ramirez, S. H., Merkel, S. F., Sena‐Esteves, M., & Breakefield, X. O. (2014) · Neurotherapeutics · doi:10.1007/s13311-014-0299-5 · Cited by 96 · see citing works
Langford-Smith, A., Wilkinson, F. L., Langford‐Smith, K., Holley, R. J., Sergijenko, A., Howe, S. J., Bennett, W. R., Jones, S. A., Wraith, J. E., Merry, C. L. R., et al. (2012) · Molecular Therapy · doi:10.1038/mt.2012.82 · Cited by 96 · see citing works
Bitzenhofer, S. H., Ahlbeck, J., Wolff, A., Wiegert, J. S., Gee, C. E., Oertner, T. G., & Hanganu‐Opatz, I. L. (2017) · Nature Communications · doi:10.1038/ncomms14563 · Cited by 92 · see citing works
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
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 · see citing works
Masamizu, Y., Okada, T., Kawasaki, K., Ishibashi, H., Yuasa, S., Takeda, S., Hasegawa, I., & Nakahara, K. (2011) · Neuroscience · doi:10.1016/j.neuroscience.2011.06.080 · Cited by 93 · 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
Differences in neurotropism and neurotoxicity among retrograde viral tracers
Sun, L., Tang, Y., Yan, K., Yu, J., Zou, Y., Xu, W., Xiao, K., Zhang, Z., Li, W., Wu, B., et al. (2019) · Molecular Neurodegeneration · doi:10.1186/s13024-019-0308-6 · Cited by 91 · see citing works
McFarland, N. R., Lee, J., Hyman, B. T., & McLean, P. J. (2009) · Journal of Neurochemistry · doi:10.1111/j.1471-4159.2009.06010.x · Cited by 91 · see citing works
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 · see citing works
Self-citationKörbelin, J., Sieber, T., Michelfelder, S., Lunding, L. P., Spies, E., Hunger, A., Alawi, M., Rapti, K., Indenbirken, D., Müller, O. J., et al. (2016) · Molecular Therapy · doi:10.1038/mt.2016.62 · Cited by 88 · see citing works
Potential of AAV vectors in the treatment of metabolic disease
Alexander, I. E., Cunningham, S. C., Logan, G. J., & Christodoulou, J. (2008) · Gene Therapy · doi:10.1038/gt.2008.64 · Cited by 88 · see citing works
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
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 84 · see citing works
Engineering Gene Therapy: Advances and Barriers
Shahryari, A., Burtscher, I., Nazari, Z., & Lickert, H. (2021) · Advanced Therapeutics · doi:10.1002/adtp.202100040 · Cited by 84 · see citing works
Friedreich Ataxia: Clinical Features and New Developments
Keita, M., McIntyre, K., Rodden, L. N., Schadt, K. A., & Lynch, D. R. (2022) · Neurodegenerative Disease Management · doi:10.2217/nmt-2022-0011 · Cited by 79 · see citing works
Systemic gene delivery to the central nervous system using Adeno-associated virus
Bourdenx, M., Dutheil, N., Bézard, E., & Dehay, B. (2014) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2014.00050 · Cited by 79 · see citing works
Alarcón-Arís, D., Pavia-Collado, R., Miquel-Rio, L., Cóppola-Segovia, V., Ferrés‐Coy, A., Ruiz‐Bronchal, E., Galofré, M., Paz, V., Campa, L., Revilla, R., et al. (2020) · EBioMedicine · doi:10.1016/j.ebiom.2020.102944 · Cited by 76 · see citing works
Franz, C. K., Federici, T., Yang, J., Backus, C., Oh, S. S., Teng, Q., Carlton, E. F., Bishop, K. M., Gasmi, M., Bartus, R. T., et al. (2008) · Neurobiology of Disease · doi:10.1016/j.nbd.2008.12.003 · Cited by 77 · see citing works
Hutson, T. H., Verhaagen, J., Yáñez‐Muñoz, R. J., & Moon, L. D. F. (2011) · Gene Therapy · doi:10.1038/gt.2011.71 · Cited by 74 · see citing works
Optical techniques in optogenetics
Mohanty, S. K., & Lakshminarayanan, V. (2015) · Journal of Modern Optics · doi:10.1080/09500340.2015.1010620 · Cited by 75 · 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
Bey, K., Ciron, C., Dubreil, L., Deniaud, J., Ledevin, M., Cristini, J., Blouin, V., Aubourg, P. R., & Colle, M.-A. (2017) · Gene Therapy · doi:10.1038/gt.2017.18 · Cited by 73 · see citing works
Harris, J. A., Wook Oh, S., & Zeng, H. (2012) · Current Protocols in Neuroscience · doi:10.1002/0471142301.ns0120s59 · Cited by 72 · see citing works
Vandenberghe, L. H., Breous, E., Nam, H.-J., Gao, G., Xiao, R., Sandhu, A., Johnston, J., Debyser, Z., Agbandje‐McKenna, M., & Wilson, J. M. (2009) · Gene Therapy · doi:10.1038/gt.2009.101 · Cited by 72 · see citing works
Self-citationRationally Engineered AAV Capsids Improve Transduction and Volumetric Spread in the CNS
Kanaan, N. M., Sellnow, R. C., Boye, S. L., Coberly, B., Bennett, A. D., Agbandje‐McKenna, M., Sortwell, C. E., Hauswirth, W. W., Boye, S. L., & Manfredsson, F. P. (2017) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2017.06.011 · Cited by 74 · see citing works
Optogenetics in the nonhuman primate
Han, X. (2012) · Progress in brain research · doi:10.1016/b978-0-444-59426-6.00011-2 · Cited by 71 · see citing works
Velde, G. V., Rangarajan, J. R., Toelen, J., Dresselaers, T., Ibrahimi, A., Krylychkina, O., Vreys, R., der Linden, A. V., Maes, F., Debyser, Z., et al. (2011) · Gene Therapy · doi:10.1038/gt.2011.2 · Cited by 71 · see citing works
Self-citationNoè, F. M., Vaghi, V., Balducci, C., Fitzsimons, H. L., Bland, R. J., Zardoni, D., Sperk, G., Carli, M., During, M. J., & Vezzani, A. (2010) · Gene Therapy · doi:10.1038/gt.2010.23 · Cited by 69 · see citing works
Survival advantage of neonatal CNS gene transfer for late infantile neuronal ceroid lipofuscinosis
Sondhi, D., Peterson, D. A., Edelstein, A., del Fierro, K., Hackett, N. R., & Crystal, R. G. (2008) · Experimental Neurology · doi:10.1016/j.expneurol.2008.04.022 · Cited by 68 · see citing works
Cunningham, J., Pivirotto, P., Bringas, J., Suzuki, B. M., Vijay, S., Sanftner, L. M., Kitamura, M., Chan, C. M., & Bankiewicz, K. S. (2008) · Molecular Therapy · doi:10.1038/mt.2008.111 · Cited by 68 · see citing works
Wu, T., Töpfer, K., Lin, S.-W., Li, H., Bian, A., Zhou, X. Y., High, K. A., & Ertl, H. C. (2012) · Molecular Therapy · doi:10.1038/mt.2011.280 · Cited by 66 · see citing works
Civiero, L., Cirnaru, M., Beilina, A. G., Rodella, U., Russo, I., Belluzzi, E., Lobbestael, E., Reyniers, L., Hondhamuni, G., Lewis, P. A., et al. (2015) · Journal of Neurochemistry · doi:10.1111/jnc.13369 · Cited by 66 · see citing works
Self-citationBlits, 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 · see citing works
3D Imaging of Axons in Transparent Spinal Cords from Rodents and Nonhuman Primates
Soderblom, C., Lee, D.-H., Dawood, A., Carballosa-Gautam, M. M., Santamaría, A. J., Benavides, F. D., Jergová, S., Grumbles, R. M., Thomas, C. K., Park, K. K., et al. (2015) · eNeuro · doi:10.1523/eneuro.0001-15.2015 · Cited by 66 · see citing works
Ivanova, E., & Pan, Z. (2009) · PubMed Central · Cited by 66 · see citing works
Albert, K., Voutilainen, M. H., Domanskyi, A., & Airavaara, M. (2017) · Genes · doi:10.3390/genes8020063 · Cited by 66 · see citing works
Autism spectrum disorder: prospects for treatment using gene therapy
Benger, M., Kinali, M., & Mazarakis, N. D. (2018) · Molecular Autism · doi:10.1186/s13229-018-0222-8 · Cited by 67 · see citing works
Fortun, J., Puzis, R., Pearse, D. D., Gage, F. H., & Bunge, M. B. (2009) · Journal of Neurotrauma · doi:10.1089/neu.2008.0807 · Cited by 64 · see citing works
Allele-Specific Silencing of Mutant Huntingtin in Rodent Brain and Human Stem Cells
Drouet, V., Ruiz, M., Zala, D., Feyeux, M., Aurégan, G., Cambon, K., Troquier, L., Carpentier, J., Aubert, S., Mérienne, N., et al. (2014) · PLoS ONE · doi:10.1371/journal.pone.0099341 · Cited by 64 · see citing works
Viral Vector-Based Models of Parkinson’s Disease
der Perren, A. V., den Haute, C. V., & Baekelandt, V. (2014) · Current topics in behavioral neurosciences · doi:10.1007/7854_2014_310 · Cited by 61 · see citing works
Self-citationZou, L., Tian, H., Guan, S., Ding, J., Gao, L., Wang, J., & Fang, Y. (2021) · Nature Communications · doi:10.1038/s41467-021-26168-0 · Cited by 60 · see citing works
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 56 · see citing works
Self-citationCiron, 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 · see citing works
Extensive Transduction and Enhanced Spread of a Modified AAV2 Capsid in the Non-human Primate CNS
Naidoo, J., Stanek, L. M., Ohno, K., Trewman, S., Samaranch, L., Hadaczek, P., O’Riordan, C. R., Sullivan, J., Sebastián, W. S., Bringas, J. R., et al. (2018) · Molecular Therapy · doi:10.1016/j.ymthe.2018.07.008 · Cited by 59 · see citing works
Henriques, A., Pitzer, C., Dittgen, T., Klugmann, M., Dupuis, L., & Schneider, A. (2010) · Molecular Therapy · doi:10.1038/mt.2010.271 · Cited by 59 · see citing works
- Authors
- Taymans, J.-M., Vandenberghe, L. H., Haute, C. V. D., Thiry, I., Deroose, C. M., Mortelmans, L., Wilson, J. M., Debyser, Z., & Baekelandt, V.
- Affiliations
- KU Leuven (BE)
- University of Pennsylvania (US)
- Universitair Ziekenhuis Leuven (BE)
- ORCID
- Venue
- Human Gene Therapy
- Publisher
- SAGE Publications
- Published
- 2007-03
- Volume
- 18
- Issue
- 3
- Pages
- 195-206
- ISSN
- 1043-0342 (ISSN-L)
- 1557-7422
- OpenAlex
- W1973418987
- Cited by
- 305
- References
- 60
- Citations by year
- 2026: 5 · 2025: 6 · 2024: 6 · 2023: 6 · 2022: 16 · 2021: 21 · 2020: 11 · 2019: 18 · 2018: 18 · 2017: 11 · 2016: 15 · 2015: 21 · 2014: 24 · 2013: 12 · 2012: 13
- FWCI
- 13.03
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Human Gene Therapy (publishedVersion)
- PubMed (publishedVersion)
- Lirias (submittedVersion)
- 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 serotypes, transduction efficiency, dose-response relationship, intrastriatal injection, intrahippocampal injection, caudate-putamen, substantia nigra, mouse brain, gene therapy vectors, disease modeling
- MeSH terms
- Dependovirus, Animals, Brain, Genetic Vectors, Humans, Mice, Inbred C57BL, Transduction, Genetic, Green Fluorescent Proteins, Mice
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
