The DOI matches its Crossref record; 42 of the 42 references it cites were found in a registry or an index.
Titration of AAV-2 particles via a novel capsid ELISA: packaging of genomes can limit production of recombinant AAV-2
Abstract
We demonstrate the rapid and reliable quantification of physical AAV-2 (adeno-associated virus type 2) particles via a novel ELISA based on a monoclonal antibody which selectively recognizes assembled AAV-2 capsids. Titration of a variety of recombinant AAV-2 (rAAV) preparations revealed that at least 80+percent of all particles were empty, compared with a maximum of 50percent in wild-type AAV-2 stocks, indicating that the recombinant genomes were less efficiently encapsidated. This finding was confirmed upon titration of CsCl gradient fractions from recombinant and wild-type AAV-2 stocks. ELISA-based measurement of capsid numbers revealed a large number of physical particles with low densities corresponding to empty capsids in the recombinant, but not in the wild-type AAV-2 preparations. Moreover, additional expression of VP proteins during rAAV production was found to result in an excessive capsid formation, whilst yielding only minor increases in DNA-containing or transducing rAAV particles. We conclude that encapsidation of viral genomes rather than capsid assembly can be limiting for rAAV production, provided that a critical level of VP expression is maintained. The feasibility of quantifying AAV-2 capsid numbers via the ELISA allows determination of physical to DNA-containing or infectious particle ratios. These are important parameters which should help to optimize and standardize the production and application of recombinant AAV-2.
Overview
Integrity signals
Registry
Cites
Statistics
Verification
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?
In the citing paper
“ELISA involves multiple lengthy incubation and washing steps prior to analysis, for a total turnaround time of 4–5 h, or up to a full working day.26,61, 62, 63”
From Analytical methods for process and product characterization of recombinant adeno-associated virus-based gene therapies (2021) · cites this paper as [61]
In the citing paper
“Specificity is high, with little cross-reactivity between serotypes, and monoclonal antibodies specific to the conformational epitope of serotypes rAAV1, -2, -3, -5, -6, -8, -9, and rh.10 are commercially available.61,62”
From Analytical methods for process and product characterization of recombinant adeno-associated virus-based gene therapies (2021) · cites this paper as [61]
In the citing paper
“The assay is typically performed after a purification step but is sufficiently robust and specific to be used on samples with moderate matrix effects and free capsid proteins, like cell lysates.26,37,61,85”
From Analytical methods for process and product characterization of recombinant adeno-associated virus-based gene therapies (2021) · cites this paper as [61]
In the citing paper
“Specificity and robustness to matrix effects are major advantages of ELISA, but in considering the long turnaround time, moderate throughput of only 10 samples per 96-well plate, and labor-intensive sample preparation, it is challenging to implement for routine quantification of capsid titer in a process development setting without automated liquid handling.”
From Analytical methods for process and product characterization of recombinant adeno-associated virus-based gene therapies (2021) · cites this paper as [61]
In the citing paper
“The quantification limit is around 108 capsid particles (cps) per milliliter, depending on serotype, and the linear range is limited to 1–2 orders of magnitude, so that pre-titration is recommended for samples of unknown titer.61,62”
From Analytical methods for process and product characterization of recombinant adeno-associated virus-based gene therapies (2021) · cites this paper as [61]
Citations
Cites
Firefly Luciferase Gene: Structure and Expression in Mammalian Cells
de Wet, J. R., Wood, K. V., DeLuca, M., Helinski, D. R., & Subramani, S. E. (1987) · Molecular and Cellular Biology · doi:10.1128/mcb.7.2.725 · Cited by 3,088
Xiao, X., Li, J., & Samulski, R. J. (1998) · Journal of Virology · doi:10.1128/jvi.72.3.2224-2232.1998 · Cited by 1,381
Self-citationKaplitt, M. G., Leone, P., Samulski, R. J., Xiao, X., Pfaff, D. W., O’Malley, K. L., & During, M. J. (1994) · Nature Genetics · doi:10.1038/ng1094-148 · Cited by 1,031
Self-citationXiao, X., Li, J., & Samulski, R. J. (1996) · Journal of Virology · doi:10.1128/jvi.70.11.8098-8108.1996 · Cited by 913
Self-citationSite-specific integration by adeno-associated virus.
Kotin, R. M., Siniscalco, M., Samulski, R. J., Zhu, X. D., Hunter, L., Laughlin, C. A., McLaughlin, S., Muzyczka, N., Rocchi, M., & Berns, K. I. (1990) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.87.6.2211 · Cited by 906
Self-citationChen, C. A., & Okayama, H. (1990) · PubMed · Cited by 898
Site-specific integration by adeno-associated virus.
Linden, R. M., Ward, P., Giraud, C., Winocour, E., & Berns, K. I. (1996) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.93.21.11288 · Cited by 805
Targeted integration of adeno‐associated virus (AAV) into human chromosome 19.
Samulski, R. J., Zhu, X., Xiao, X., Brook, J. D., Housman, D. E., Epstein, N., & Hunter, L. A. (1991) · The EMBO Journal · doi:10.1002/j.1460-2075.1991.tb04964.x · Cited by 783
Self-citationEvaluation of the concentration and bioactivity of adenovirus vectors for gene therapy
Mittereder, N., March, K. L., & Trapnell, B. C. (1996) · Journal of Virology · doi:10.1128/jvi.70.11.7498-7509.1996 · Cited by 778
Ferrari, F. K., Samulski, T., Shenk, T., & Samulski, R. J. (1996) · Journal of Virology · doi:10.1128/jvi.70.5.3227-3234.1996 · Cited by 761
Self-citation
Research works printed but not linked: 1
- Rittner K, Stoppler H, Pawlita M, Sczakiel G . Versatile eucaryotic vectors for strong and constitutive transient and stable gene expression Meth Mol Cell Biol 1991 2: 176–181
Cited by
Sorted by citations
AAV-Mediated Gene Therapy for Research and Therapeutic Purposes
Samulski, R. J., & Muzyczka, N. (2014) · Annual Review of Virology · doi:10.1146/annurev-virology-031413-085355 · Cited by 609
Self-citationAurnhammer, C., Haase, M., Müther, N., Mück-Häusl, M., Rauschhuber, C., Huber, I., Nitschko, H., Busch, U., Sing, A., Ehrhardt, A., et al. (2012) · Human Gene Therapy Methods · doi:10.1089/hgtb.2011.034 · Cited by 494
Müller, O. J., Kaul, F., Weitzman, M. D., Pasqualini, R., Arap, W., Kleinschmidt, J. A., & Trepel, M. (2003) · Nature Biotechnology · doi:10.1038/nbt856 · Cited by 411
Self-citationWu, P., Xiao, W. S., Conlon, T. J., Hughes, J. A., Agbandje‐McKenna, M., Ferkol, T. W., Flotte, T. R., & Muzyczka, N. (2000) · Journal of Virology · doi:10.1128/jvi.74.18.8635-8647.2000 · Cited by 396
A viral assembly factor promotes AAV2 capsid formation in the nucleolus
Sonntag, F., Schmidt, K., & Kleinschmidt, J. A. (2010) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1001673107 · Cited by 392
Self-citationIdentification of a heparin-binding motif on adeno-associated virus type 2 capsids
Kern, A., Schmidt, K., Leder, C., Müller, O. J., Wobus, C. E., Bettinger, K., Von der Lieth, C. W., King, J. A., & Kleinschmidt, J. A. (2003) · Journal of Virology · doi:10.1128/jvi.77.20.11072-11081.2003 · Cited by 380
Self-citationGrimm, D., & Kay, M. A. (2003) · Current Gene Therapy · doi:10.2174/1566523034578285 · Cited by 351
Self-citationGrimm, D., Kay, M. A., & Kleinschmidt, J. A. (2003) · Molecular Therapy · doi:10.1016/s1525-0016(03)00095-9 · Cited by 344
Self-citationGirod, A., Wobus, C. E., Zádori, Z., Ried, M., Leike, K., Tijssen, P., Kleinschmidt, J. A., & Hallek, M. (2002) · Journal of General Virology · doi:10.1099/0022-1317-83-5-973 · Cited by 354
Self-citationDNA helicase‐mediated packaging of adeno‐associated virus type 2 genomes into preformed capsids
King, J. A., Dubielzig, R., Grimm, D., & Kleinschmidt, J. A. (2001) · The EMBO Journal · doi:10.1093/emboj/20.12.3282 · Cited by 338
Self-citationAAV vectors: is clinical success on the horizon?
Monahan, P. E., & Samulski, R. J. (2000) · Gene Therapy · doi:10.1038/sj.gt.3301109 · Cited by 291
Self-citationSonntag, F., Bleker, S., Leuchs, B., Fischer, R., & Kleinschmidt, J. A. (2006) · Journal of Virology · doi:10.1128/jvi.01056-06 · Cited by 297
Self-citationWobus, C. E., Hügle-Dörr, B., Girod, A., Petersen, G., Hallek, M., & Kleinschmidt, J. A. (2000) · Journal of Virology · doi:10.1128/jvi.74.19.9281-9293.2000 · Cited by 273
Self-citationIntracellular transport of recombinant adeno-associated virus vectors
Nonnenmacher, M., & Weber, T. (2012) · Gene Therapy · doi:10.1038/gt.2012.6 · Cited by 258
Hepatocyte Growth Factor Receptor Is a Coreceptor for Adeno-Associated Virus Type 2 Infection
Kashiwakura, Y., Tamayose, K., Iwabuchi, K., Hirai, Y., Shimada, T., Matsumoto, K., Nakamura, T., Watanabe, M., Oshimi, K., & Daida, H. (2005) · Journal of Virology · doi:10.1128/jvi.79.1.609-614.2005 · Cited by 246
Quantification of adeno-associated virus particles and empty capsids by optical density measurement
Sommer, J. Ü. M., Smith, P. H., Parthasarathy, S., Isaacs, J., Vijay, S., Kieran, J., Powell, S. K., McClelland, A., & Wright, J. F. (2003) · Molecular Therapy · doi:10.1016/s1525-0016(02)00019-9 · Cited by 230
AAV Capsid Structure and Cell Interactions
Agbandje-McKenna, M., & Kleinschmidt, J. (2012) · Methods in Molecular Biology · doi:10.1007/978-1-61779-370-7_3 · Cited by 215
Self-citationWarrington, K. H., Gorbatyuk, O. S., Harrison, J. K., Opie, S. R., Zolotukhin, S., & Muzyczka, N. (2004) · Journal of Virology · doi:10.1128/jvi.78.12.6595-6609.2004 · Cited by 205
Bleker, S., Sonntag, F., & Kleinschmidt, J. A. (2005) · Journal of Virology · doi:10.1128/jvi.79.4.2528-2540.2005 · Cited by 200
Self-citationKronenberg, S., Böttcher, B., von der Lieth, C. W., Bleker, S., & Kleinschmidt, J. A. (2005) · Journal of Virology · doi:10.1128/jvi.79.9.5296-5303.2005 · Cited by 198
Self-citationGimpel, A. L., Katsikis, G., Sha, S., Maloney, A. J., Hong, M. S., Nguyen, T. N. T., Wolfrum, J., Springs, S. L., Sinskey, A. J., Manalis, S. R., et al. (2021) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2021.02.010 · Cited by 185
Sonntag, F., Köther, K., Schmidt, K., Weghofer, M., Raupp, C., Nieto, K., Kuck, A., Gerlach, B., Böttcher, B., Müller, O. J., et al. (2011) · Journal of Virology · doi:10.1128/jvi.05359-11 · Cited by 184
Self-citationPharmacology of Recombinant Adeno-associated Virus Production
Penaud-Budloo, M., François, A., Clément, N., & Ayuso, E. (2018) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2018.01.002 · Cited by 177
Shi, W., Arnold, G. S., & Bartlett, J. S. (2001) · Human Gene Therapy · doi:10.1089/104303401750476212 · Cited by 167
Paulk, N. K., Pekrun, K., Zhu, E., Nygaard, S., Li, B., Xu, J., Chu, K., Leborgne, C., Dane, A. P., Haft, A., et al. (2018) · Molecular Therapy · doi:10.1016/j.ymthe.2017.09.021 · Cited by 161
Electron cryo‐microscopy and image reconstruction of adeno‐associated virus type 2 empty capsids
Kronenberg, S., Kleinschmidt, J. A., & Böttcher, B. (2001) · The EMBO Reports · doi:10.1093/embo-reports/kve234 · Cited by 161
Self-citationQu, G., Bahr-Davidson, J., Prado, J., Tai, A., Cataniag, F., McDonnell, J., Zhou, J.-M., Hauck, B., Luna, J., Sommer, J. M., et al. (2006) · Journal of Virological Methods · doi:10.1016/j.jviromet.2006.11.019 · Cited by 156
Müller, O. J., Leuchs, B., Pleger, S. T., Grimm, D., Franz, W. M., Katus, H. A., & Kleinschmidt, J. A. (2006) · Cardiovascular Research · doi:10.1016/j.cardiores.2005.12.017 · Cited by 156
Self-citationLux, K., Goerlitz, N., Schlemminger, S., Perabò, L., Goldnau, D., Endell, J., Leike, K., Kofler, D. M., Finke, S., Hallek, M. J., et al. (2005) · Journal of Virology · doi:10.1128/jvi.79.18.11776-11787.2005 · Cited by 159
Combinatorial engineering of a gene therapy vector: directed evolution of adeno‐associated virus
Perabo, L., Endell, J., King, S., Lux, K., Goldnau, D., Hallek, M., & Büning, H. (2006) · The Journal of Gene Medicine · doi:10.1002/jgm.849 · Cited by 136
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 138
Ried, M. U., Girod, A., Leike, K., Büning, H., & Hallek, M. J. (2002) · Journal of Virology · doi:10.1128/jvi.76.9.4559-4566.2002 · Cited by 132
Benkhelifa‐Ziyyat, S., Besse, A., Roda, M., Duqué, S. I., Astord, S., Carcenac, R., Marais, T., & Barkats, M. (2013) · Molecular Therapy · doi:10.1038/mt.2012.261 · Cited by 128
Receptor targeting of adeno-associated virus vectors
Büning, H., Ried, M. U., Perabò, L., Gerner, F., Huttner, N. A., Enssle, J., & Hallek, M. J. (2003) · Gene Therapy · doi:10.1038/sj.gt.3301976 · Cited by 124
Huttner, N. A., Girod, A., Perabo, L., Edbauer, D., Kleinschmidt, J. A., Büning, H., & Hallek, M. (2003) · Gene Therapy · doi:10.1038/sj.gt.3302123 · Cited by 124
Self-citationTransient transfection methods for clinical adeno-associated viral vector production
Wright, J. F. (2009) · Human Gene Therapy · doi:10.1089/hum.2009.064 · Cited by 131
Intracellular trafficking of adeno-associated virus (AAV) vectors: challenges and future directions
Riyad, J. M., & Weber, T. (2021) · Gene Therapy · doi:10.1038/s41434-021-00243-z · Cited by 124
Production methods for gene transfer vectors based on adeno-associated virus serotypes
Grimm, D. (2002) · Methods · doi:10.1016/s1046-2023(02)00219-0 · Cited by 117
Self-citationGrimm, D., Pandey, K., Nakai, H., Storm, T. A., & Kay, M. A. (2006) · Journal of Virology · doi:10.1128/jvi.80.1.426-439.2006 · Cited by 118
Self-citationPopa-Wagner, R., Porwal, M., Kann, M., Reuss, M., Weimer, M., Florin, L., & Kleinschmidt, J. A. (2012) · Journal of Virology · doi:10.1128/jvi.00282-12 · Cited by 109
Self-citationVeldwijk, M. R., Topaly, J., Laufs, S., Hengge, U. R., Wenz, F. K., Zeller, W. J., & Früehauf, S. (2002) · Molecular Therapy · doi:10.1006/mthe.2002.0659 · Cited by 106
Zhao, H., Lee, K.-J., Daris, M., Lin, Y., Wolfe, T., Sheng, J., Plewa, C., Wang, S., & Meisen, W. H. (2020) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2020.06.004 · Cited by 98
Nanoparticle number concentration measurements by multi-angle dynamic light scattering
Austin, J., Minelli, C., Hamilton, D., Wywijas, M., & Jones, H. J. (2020) · Journal of nanoparticle research · doi:10.1007/s11051-020-04840-8 · Cited by 88
Current issues in adeno-associated viral vector production
Merten, O.-W., Gény-Fiamma, C., & Douar, A. M. (2005) · Gene Therapy · doi:10.1038/sj.gt.3302615 · Cited by 101
Lock, M., Alvira, M. R., & Wilson, J. M. (2012) · Human Gene Therapy Methods · doi:10.1089/hgtb.2011.217 · Cited by 99
Production, Processing, and Characterization of Synthetic AAV Gene Therapy Vectors
Andari, J. E., & Grimm, D. (2020) · Biotechnology Journal · doi:10.1002/biot.202000025 · Cited by 105
Self-citationAyuso, 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
Self-citationMapping a Neutralizing Epitope onto the Capsid of Adeno-Associated Virus Serotype 8
Gurda, B. L., Raupp, C., Popa-Wagner, R., Naumer, M., Olson, N. H., Ng, R., McKENNA, R. V., Baker, T. S., Kleinschmidt, J. A., & Agbandje‐McKenna, M. (2012) · Journal of Virology · doi:10.1128/jvi.00218-12 · Cited by 97
Self-citationProperties of the Adeno-Associated Virus Assembly-Activating Protein
Naumer, M., Sonntag, F., Schmidt, K., Nieto, K., Panke, C., Davey, N. E., Popa-Wagner, R., & Kleinschmidt, J. A. (2012) · Journal of Virology · doi:10.1128/jvi.01675-12 · Cited by 97
Self-citationManufacturing of recombinant adeno-associated viruses using mammalian expression platforms
Robert, M., Chahal, P. S., Audy, A., Kamen, A., Gilbert, R., & Gaillet, B. (2017) · Biotechnology Journal · doi:10.1002/biot.201600193 · Cited by 89
Brument, N., Morenweiser, R., Blouin, V., Toublanc, E., Raimbaud, I., Cherel, Y., Folliot, S., Gaden, F., BOULANGER, P., KRONERLUX, G., et al. (2002) · Molecular Therapy · doi:10.1016/s1525-0016(02)90719-7 · Cited by 86
Fu, X., Chen, W.-C., Argento, C., Clarner, P., Bhatt, V., Dickerson, R., Bou-Assaf, G., Bakhshayeshi, M., Lu, X., Bergelson, S., et al. (2019) · Human Gene Therapy Methods · doi:10.1089/hgtb.2019.088 · Cited by 77
Martin, J. M., Frederick, A., Luo, Y., Jackson, R., Joubert, M., Sol, B., Poulin, F., Pastor, E., Armentano, D., Wadsworth, S. C., et al. (2013) · Human Gene Therapy Methods · doi:10.1089/hgtb.2013.046 · Cited by 79
Adeno-Associated Virus Vectors for Short Hairpin RNA Expression
Grimm, D., Pandey, K., & Kay, M. A. (2005) · Methods in enzymology on CD-ROM/Methods in enzymology · doi:10.1016/s0076-6879(04)92023-x · Cited by 82
Self-citationSchnödt, M., & Büning, H. (2017) · Human Gene Therapy Methods · doi:10.1089/hgtb.2016.188 · Cited by 77
Development of AAV serotype-specific ELISAs using novel monoclonal antibodies
Kuck, D., Kern, A., & Kleinschmidt, J. A. (2007) · Journal of Virological Methods · doi:10.1016/j.jviromet.2006.10.005 · Cited by 74
Self-citationMichelfelder, S., Kohlschütter, J., Skorupa, A., Pfennings, S., Müller, O. J., Kleinschmidt, J. A., & Trepel, M. (2009) · PLoS ONE · doi:10.1371/journal.pone.0005122 · Cited by 75
Self-citationNear-perfect infectivity of wild-type AAV as benchmark for infectivity of recombinant AAV vectors
Zeltner, N., Kohlbrenner, E., Clément, N., Weber, T., & Linden, R. M. (2010) · Gene Therapy · doi:10.1038/gt.2010.27 · Cited by 75
Isolation of targeted AAV2 vectors from novel virus display libraries
Waterkamp, D. A., Müller, O. J., Ying, Y., Trepel, M., & Kleinschmidt, J. A. (2006) · The Journal of Gene Medicine · doi:10.1002/jgm.967 · Cited by 69
Self-citationBleker, S., Pawlita, M., & Kleinschmidt, J. A. (2006) · Journal of Virology · doi:10.1128/jvi.80.2.810-820.2006 · Cited by 75
Self-citationAdeno-Associated Virus Genome Interactions Important for Vector Production and Transduction
Maurer, A. C., & Weitzman, M. D. (2020) · Human Gene Therapy · doi:10.1089/hum.2020.069 · Cited by 67
Critical assessment of current adeno-associated viral vector production and quantification methods
Aucoin, M. G., Perrier, M., & Kamen, A. (2007) · Biotechnology Advances · doi:10.1016/j.biotechadv.2007.09.001 · Cited by 65
Kuck, D., Lau, T., Leuchs, B., Kern, A., Müller, M., Gissmann, L., & Kleinschmidt, J. A. (2006) · Journal of Virology · doi:10.1128/jvi.80.6.2621-2630.2006 · Cited by 66
Self-citationMichelfelder, S., Lee, M., deLima-Hahn, E., Wilmes, T., Kaul, F., Müller, O. J., Kleinschmidt, J. A., & Trepel, M. (2007) · Experimental Hematology · doi:10.1016/j.exphem.2007.07.018 · Cited by 65
Self-citationJin, X., Liu, L., Nass, S., O’Riordan, C. R., Pastor, E., & Zhang, X. K. (2017) · Human Gene Therapy Methods · doi:10.1089/hgtb.2016.178 · Cited by 62
Production of clinical-grade recombinant adeno-associated virus vectors
Snyder, R. O., & Flotte, T. R. (2002) · Current Opinion in Biotechnology · doi:10.1016/s0958-1669(02)00369-5 · Cited by 58
Fakhiri, J., Schneider, M. A., Puschhof, J., Stanifer, M. L., Schildgen, V., Holderbach, S., Voß, Y., Andari, J. E., Schildgen, O., Boulant, S., et al. (2019) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2019.01.003 · Cited by 58
Self-citationGalibert, L., & Merten, O. (2011) · Journal of Invertebrate Pathology · doi:10.1016/j.jip.2011.05.008 · Cited by 61
Aucoin, M. G., Perrier, M., & Kamen, A. (2006) · Biotechnology and Bioengineering · doi:10.1002/bit.21069 · Cited by 56
Cao, L., Liu, Y., During, M. J., & Xiao, W. (2000) · Journal of Virology · doi:10.1128/jvi.74.24.11456-11463.2000 · Cited by 56
Bouças, J., Lux, K., Huber, A., Schievenbusch, S., von Freyend, M. J., Perabò, L., Quadt‐Humme, S., Odenthal, M., Hallek, M. J., & Büning, H. (2009) · The Journal of Gene Medicine · doi:10.1002/jgm.1392 · Cited by 57
Scalable production of adeno‐associated virus type 2 vectors via suspension transfection
Park, J. Y., Lim, B., Lee, K., Kim, Y. K., & Jo, E. (2006) · Biotechnology and Bioengineering · doi:10.1002/bit.20776 · Cited by 55
Werling, N. J., Satkunanathan, S., Thorpe, R., & Zhao, Y. (2015) · Human Gene Therapy Methods · doi:10.1089/hgtb.2015.013 · Cited by 53
Identification of the heparin binding site on adeno-associated virus serotype 3B (AAV-3B)
Lerch, T. F., & Chapman, M. S. (2011) · Virology · doi:10.1016/j.virol.2011.10.007 · Cited by 55
Characterization of AAV vectors: a review of analytical techniques and critical quality attributes
Kontogiannis, T., Braybrook, J., McElroy, C., Foy, C., Whale, A. S., Quaglia, M., & Smales, C. M. (2024) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2024.101309 · Cited by 57
Chahal, P. S., Aucoin, M. G., & Kamen, A. (2006) · Journal of Virological Methods · doi:10.1016/j.jviromet.2006.09.011 · Cited by 52
Yoon-Robarts, M., Blouin, A. G., Bleker, S., Kleinschmidt, J. A., Aggarwal, A. K., Escalante, C. R., & Linden, R. M. (2004) · Journal of Biological Chemistry · doi:10.1074/jbc.m403900200 · Cited by 52
Self-citationRecent advances in recombinant adeno-associated virus vector production
Clark, K. R. (2002) · Kidney International · doi:10.1046/j.1523-1755.2002.0610s1009.x · Cited by 52
Optimization of stealth adeno-associated virus vectors by randomization of immunogenic epitopes
Maersch, S., Huber, A., Büning, H., Hallek, M., & Perabo, L. (2010) · Virology · doi:10.1016/j.virol.2009.10.021 · Cited by 50
Rey‐Rico, A., Venkatesan, J. K., Frisch, J., Schmitt, G., Monge‐Marcet, A., López‐Chicón, P., Mata, Á., Semino, C. E., Madry, H., & Cucchiarini, M. (2015) · Acta Biomaterialia · doi:10.1016/j.actbio.2015.02.013 · Cited by 51
Structural and cellular biology of adeno-associated virus attachment and entry
Zengel, J. R., & Carette, J. E. (2020) · Advances in virus research · doi:10.1016/bs.aivir.2020.01.002 · Cited by 47
Transfection-free and scalable recombinant AAV vector production using HSV/AAV hybrids
Booth, M. J., Mistry, A., Li, X., Thrasher, A. J., & Coffin, R. S. (2004) · Gene Therapy · doi:10.1038/sj.gt.3302226 · Cited by 44
In vivo gene delivery for development of mammalian models for Parkinson's disease
Ulusoy, A., Björklund, T., Hermening, S., & Kirik, D. (2007) · Experimental Neurology · doi:10.1016/j.expneurol.2007.09.011 · Cited by 42
Construction of an rAAV Producer Cell Line through Synthetic Biology
Lee, Z., Lu, M., Irfanullah, E., Soukup, M., & Hu, W. (2022) · ACS Synthetic Biology · doi:10.1021/acssynbio.2c00207 · Cited by 43
DNA Minicircle Technology Improves Purity of Adeno-associated Viral Vector Preparations
Schnödt, M., Schmeer, M., Kracher, B., Krüsemann, C., Espinosa, L. E., Grünert, A., Fuchsluger, T., Rischmüller, A., Schleef, M., & Büning, H. (2016) · Molecular Therapy - Nucleic Acids · doi:10.1038/mtna.2016.60 · Cited by 39
Development of Stable Packaging and Producer Cell Lines for the Production of AAV Vectors
Merten, O. (2024) · Microorganisms · doi:10.3390/microorganisms12020384 · Cited by 41
Su, W., Patrício, M. I., Duffy, M. R., Krakowiak, J. M., Seymour, L. W., & Cawood, R. (2022) · Nature Communications · doi:10.1038/s41467-022-28738-2 · Cited by 40
The ice age – A review on formulation of Adeno-associated virus therapeutics
Grossen, P., Skaripa-Koukelli, I., van Haasteren, J., Machado, A. H. E., & Dürr, C. (2023) · European Journal of Pharmaceutics and Biopharmaceutics · doi:10.1016/j.ejpb.2023.07.002 · Cited by 41
Kohlbrenner, E., Henckaerts, E., Rapti, K., Gordon, R. E., Linden, R. M., Hajjar, R. J., & Weber, T. (2012) · Human Gene Therapy Methods · doi:10.1089/hgtb.2012.049 · Cited by 36
Tullis, G. E., & Shenk, T. E. (2000) · Journal of Virology · doi:10.1128/jvi.74.24.11511-11521.2000 · Cited by 38
Vector characterization methods for quality control testing of recombinant adeno-associated viruses
Wright, J. F., & Zelenaia, O. (2011) · Methods in Molecular Biology · doi:10.1007/978-1-61779-095-9_11 · Cited by 34
Quantitative real-time PCR for titration of infectious recombinant AAV-2 particles
Rohr, U., Heyd, F., Neukirchen, J., Wulf, M.-A., Queitsch, I., Kroner‐Lux, G., Steidl, U. G., Fenk, R. R., Haas, R. R., & Kronenwett, R. (2005) · Journal of Virological Methods · doi:10.1016/j.jviromet.2005.03.006 · Cited by 34
Adeno-associated Virus and AAV Vectors for Gene Delivery
Carter, B. J., Burstein, H., & Peluso, R. W. (2000) · CRC Press eBooks · doi:10.1201/9781420002126-12 · Cited by 34
Shi, X., Fang, G., Shi, W., & Bartlett, J. S. (2006) · Human Gene Therapy · doi:10.1089/hum.2006.17.353 · Cited by 33
Vliet, K. V., Mohiuddin, Y., McClung, S. H., Blouin, V., Rolling, F., Moullier, P., Agbandje‐McKenna, M., & Snyder, R. O. (2009) · Journal of Virological Methods · doi:10.1016/j.jviromet.2009.03.020 · Cited by 33
Fakhiri, J., & Grimm, D. (2021) · Molecular Therapy · doi:10.1016/j.ymthe.2021.04.005 · Cited by 29
Self-citationToublanc, E., Benraiss, A., Bonnin, D., Blouin, V., Brument, N., Cartier, N., Epstein, A. L., Moullier, P., & Salvetti, A. (2004) · The Journal of Gene Medicine · doi:10.1002/jgm.542 · Cited by 29
Cell Culture Processes for the Production of Viral Vectors for Gene Therapy Purposes
Warnock, J. N., Merten, O., & Al‐Rubeai, M. (2006) · Cytotechnology · doi:10.1007/s10616-005-5507-z · Cited by 28
Quantification of Adeno-Associated Virus with Safe Nucleic Acid Dyes
Xu, J., DeVries, S. H., & Zhu, Y. (2020) · Human Gene Therapy · doi:10.1089/hum.2020.063 · Cited by 25
Adeno-associated virus (AAV) capsid engineering in liver-directed gene therapy
Rodríguez-Márquez, E., Meumann, N., & Büning, H. (2020) · Expert Opinion on Biological Therapy · doi:10.1080/14712598.2021.1865303 · Cited by 29
Metadata
- Authors
- Grimm, D., Kern, A., Pawlita, M., Ferrari, F. K., Samulski, R. J., & Kleinschmidt, J. A.
- Affiliations
- German Cancer Research Center (DE)
- Princeton University (US)
- University of North Carolina at Chapel Hill (US)
- Venue
- Gene Therapy
- Published
- 1999-07
- Volume
- 6
- Issue
- 7
- Pages
- 1322-1330
- ISSN
- 0969-7128 (ISSN-L)
- 1476-5462
- OpenAlex
- W2079090019
- Cited by
- 195
- References
- 42
- Citations by year
- 2026: 5 · 2025: 6 · 2024: 9 · 2023: 8 · 2022: 3 · 2021: 10 · 2020: 9 · 2019: 5 · 2018: 5 · 2017: 4 · 2016: 3 · 2015: 2 · 2014: 5 · 2013: 6 · 2012: 13
- FWCI
- 3.53
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Gene Therapy (publishedVersion)
- PubMed (publishedVersion)
- Funders
- Bundesministerium für Bildung und Forschung
- 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 › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Viral Infectious Diseases and Gene Expression in Insects
- Keywords
- adeno-associated virus type 2, empty capsids, capsid assembly, viral vector production, monoclonal antibodies
- MeSH terms
- Dependovirus, Antibodies, Monoclonal, Capsid, Enzyme-Linked Immunosorbent Assay, Genetic Engineering, Genetic Vectors, Humans, Genetic Therapy
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
