Cited by 344Virus-based gene therapy researchRNA Interference and Gene DeliveryViral Infectious Diseases and Gene Expression in Insects

The DOI matches its Crossref record; 34 of the 36 references it cites were found in a registry or an index.

Helper virus-free, optically controllable, and two-plasmid-based production of adeno-associated virus vectors of serotypes 1 to 6

Open access

Grimm, Dirk, Kay, Mark A, Kleinschmidt, Juergen A·2003·Molecular Therapy

DOI 10.1016/s1525-0016(03)00095-9

Open this paper in CoChat — free account Open PDF

Abstract

We present a simple and safe strategy for producing high-titer adeno-associated virus (AAV) vectors derived from six different AAV serotypes (AAV-1 to AAV-6). The method, referred to as "HOT," is helper virus free, optically controllable, and based on transfection of only two plasmids, i.e., an AAV vector construct and one of six novel AAV helper plasmids. The latter were engineered to carry AAV serotype rep and cap genes together with adenoviral helper functions, as well as unique fluorescent protein expression cassettes, allowing confirmation of successful transfection and identification of the transfected plasmid. Cross-packaging of vector DNA derived from AAV-2, -3, or -6 was up to 10-fold more efficient using our novel plasmids, compared to a conservative adenovirus-dependent method. We also identified a variety of useful antibodies, allowing detection of Rep or VP proteins, or assembled capsids, of all six AAV serotypes. Finally, we describe unique cell tropisms and kinetics of transgene expression for AAV serotype vectors in primary or transformed cells from four different species. In sum, the HOT strategy and the antibodies presented here, together with the reported findings, should facilitate and support the further development of AAV serotype vectors as powerful new tools for human gene therapy.

The claims and statistics of this paper haven’t been checked yet.

Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.

Integrity signals

Registry

Crossref · 10.1016/s1525-0016(03)00095-9

Cites

34 of 34 references verified0 citation errors · 0 not checkable

Statistics

Claims the paper makes

The claims and statistics of this paper haven’t been checked yet.

Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.

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?

Self-check

Percentages, sums and reported differences re-computed from the paper’s own numbers.

The self-check needs the paper’s full text.

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
Membrane-Associated Heparan Sulfate Proteoglycan Is a Receptor for Adeno-Associated Virus Type 2 Virions
Summerford, C., & Samulski, R. J. (1998) · Journal of Virology · doi:10.1128/jvi.72.2.1438-1445.1998 · Cited by 1,409
Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield
Zolotukhin, S., Byrne, B. J., Mason, E. G., Zolotukhin, I., Potter, M., Chesnut, K. A., Summerford, C., Samulski, R. J., & Muzyczka, N. (1999) · Gene Therapy · doi:10.1038/sj.gt.3300938 · Cited by 1,321
Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector
Kay, M. A., Manno, C. S., Ragni, M. V., Larson, P. J., Couto, L. B., McClelland, A., Glader, B. E., Chew, A. K., Tai, S. J., Herzog, R. W., et al. (2000) · Nature Genetics · doi:10.1038/73464 · Cited by 1,019
Self-citation
Cross-Packaging of a Single Adeno-Associated Virus (AAV) Type 2 Vector Genome into Multiple AAV Serotypes Enables Transduction with Broad Specificity
Rabinowitz, J. E., Rolling, F., Li, C., Conrath, H., Xiao, W., Xiao, X., & Samulski, R. J. (2002) · Journal of Virology · doi:10.1128/jvi.76.2.791-801.2002 · Cited by 762
Novel Tools for Production and Purification of Recombinant Adenoassociated Virus Vectors
Grimm, D., Kern, A., Rittner, K., & Kleinschmidt, J. A. (1998) · Human Gene Therapy · doi:10.1089/hum.1998.9.18-2745 · Cited by 701
Self-citation
A "humanized" green fluorescent protein cDNA adapted for high-level expression in mammalian cells
Zolotukhin, S., Potter, M., Hauswirth, W. W., Guy, J., & Muzyczka, N. (1996) · Journal of Virology · doi:10.1128/jvi.70.7.4646-4654.1996 · Cited by 609
Gene therapy vectors based on adeno-associated virus type 1
Xiao, W., Chirmule, N., Berta, S. C., McCullough, B., Gao, G., & Wilson, J. M. (1999) · Journal of Virology · doi:10.1128/jvi.73.5.3994-4003.1999 · Cited by 541
Recombinant adeno-associated virus type 2, 4, and 5 vectors: Transduction of variant cell types and regions in the mammalian central nervous system
Davidson, B. L., Stein, C. S., Heth, J. A., Martins, I., Kotin, R. M., Derksen, T. A., Zabner, J., Ghodsi, A., & Chiorini, J. A. (2000) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.97.7.3428 · Cited by 531
Current protocols in human genetics
Bates, G. P. (1995) · Trends in Genetics · doi:10.1016/s0168-9525(00)89147-7 · Cited by 484

Show all 36 references

Sorted by citations

Adeno-associated Virus Serotypes: Vector Toolkit for Human Gene Therapy
Wu, Z., Asokan, A., & Samulski, R. J. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2006.05.009 · Cited by 957
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
The 37/67-Kilodalton Laminin Receptor Is a Receptor for Adeno-Associated Virus Serotypes 8, 2, 3, and 9
Akache, B., Grimm, D., Pandey, K., Yant, S. R., Xu, H., & Kay, M. A. (2006) · Journal of Virology · doi:10.1128/jvi.00878-06 · Cited by 448
Self-citation
Massive accumulation of luminal protease-deficient axonal lysosomes at Alzheimer’s disease amyloid plaques
Gowrishankar, S., Yuan, P., Wu, Y., Schrag, M., Paradise, S., Grutzendler, J., De Camilli, P., & Ferguson, S. M. (2015) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1510329112 · Cited by 406
Biophysical constraints of optogenetic inhibition at presynaptic terminals
Mahn, M., Prigge, M., Ron, S., Levy, R., & Yizhar, O. (2016) · Nature Neuroscience · doi:10.1038/nn.4266 · Cited by 360
siRNA-based approaches in cancer therapy
Devi, G. R. (2006) · Cancer Gene Therapy · doi:10.1038/sj.cgt.7700931 · Cited by 359
From Virus Evolution to Vector Revolution: Use of Naturally Occurring Serotypes of Adeno-associated Virus (AAV) as Novel Vectors for Human Gene Therapy
Grimm, D., & Kay, M. A. (2003) · Current Gene Therapy · doi:10.2174/1566523034578285 · Cited by 351
Self-citation
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
Recombinant Adeno-Associated Virus Gene Therapy in Light of Luxturna (and Zolgensma and Glybera): Where Are We, and How Did We Get Here?
Keeler, A. M., & Flotte, T. R. (2019) · Annual Review of Virology · doi:10.1146/annurev-virology-092818-015530 · Cited by 322
Optical activation of lateral amygdala pyramidal cells instructs associative fear learning
Johansen, J. P., Hamanaka, H., Monfils, M. H., Behnia, R., Deisseroth, K., Blair, H. T., & LeDoux, J. E. (2010) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1002418107 · Cited by 308
High-efficiency optogenetic silencing with soma-targeted anion-conducting channelrhodopsins
Mahn, M., Gibor, L., Patil, P., Malina, K. C.-K., Oring, S., Printz, Y., Levy, R., Lampl, I., & Yizhar, O. (2018) · Nature Communications · doi:10.1038/s41467-018-06511-8 · Cited by 279
Classical transient receptor potential channel 6 (TRPC6) is essential for hypoxic pulmonary vasoconstriction and alveolar gas exchange
Weißmann, N., Dietrich, A., Fuchs, B., Kalwa, H., Ay, M., Dumitrascu, R., Olschewski, A., Storch, U., y Schnitzler, M. M., Ghofrani, H. A., et al. (2006) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0606728103 · Cited by 304
Dimensions of a projection column and architecture of VPM and POm axons in rat vibrissal cortex
Wimmer, V. C., Bruno, R. M., de Kock, C. P. J., Kuner, T., & Sakmann, B. (2010) · Cerebral Cortex · doi:10.1093/cercor/bhq068 · Cited by 263
Tmc gene therapy restores auditory function in deaf mice
Askew, C., Rochat, C., Pan, B., Asai, Y., Ahmed, H., Child, E., Schneider, B. L., Aebischer, P., & Holt, J. R. (2015) · Science Translational Medicine · doi:10.1126/scitranslmed.aab1996 · Cited by 261
A brain microvasculature endothelial cell‐specific viral vector with the potential to treat neurovascular and neurological diseases
Körbelin, J., Dogbevia, G., Michelfelder, S., Ridder, D. A., Hunger, A., Wenzel, J., Seismann, H., Lampe, M., Bannach, J., Pasparakis, M., et al. (2016) · EMBO Molecular Medicine · doi:10.15252/emmm.201506078 · Cited by 253
Self-citation
Transfection of mammalian cells using linear polyethylenimine is a simple and effective means of producing recombinant adeno-associated virus vectors
Reed, S. E., Staley, E. M., Mayginnes, J. P., Pintel, D. J., & Tullis, G. E. (2006) · Journal of Virological Methods · doi:10.1016/j.jviromet.2006.07.024 · Cited by 251
Robust cardiomyocyte-specific gene expression following systemic injection of AAV: in vivo gene delivery follows a Poisson distribution
Prasad, K.-M. R., Xu, Y., Yang, Z., Acton, S. T., & French, B. A. (2010) · Gene Therapy · doi:10.1038/gt.2010.105 · Cited by 249
FATP2 is a hepatic fatty acid transporter and peroxisomal very long-chain acyl-CoA synthetase
Falcon, A., Doege, H., Fluitt, A. H., Tsang, B., Watson, N., Kay, M. A., & Stahl, A. (2010) · American Journal of Physiology-Endocrinology and Metabolism · doi:10.1152/ajpendo.00226.2010 · Cited by 266
Self-citation
Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy
Grimm, D., Zhou, S., Nakai, H., Thomas, C. E., Storm, T. A., Fuess, S., Matsushita, T., Allen, J. M., Surosky, R. T., Lochrie, M. A., et al. (2003) · Blood · doi:10.1182/blood-2003-02-0495 · Cited by 208
Self-citation
Promoters and serotypes: targeting of adeno‐associated virus vectors for gene transfer in the rat central nervous system in vitro and in vivo
Шевцова, З. І., Malik, I., Michel, U., Bähr, M., & Kügler, S. (2004) · Experimental Physiology · doi:10.1113/expphysiol.2004.028159 · Cited by 207
Correction of human phospholamban R14del mutation associated with cardiomyopathy using targeted nucleases and combination therapy
Karakikes, I., Stillitano, F., Nonnenmacher, M. E., Tzimas, C., Sanoudou, D., Termglinchan, V., Kong, C., Rushing, S. N., Hansen, J., Ceholski, D. K., et al. (2015) · Nature Communications · doi:10.1038/ncomms7955 · Cited by 206
Phosphorylation Does Not Prompt, Nor Prevent, the Formation of -synuclein Toxic Species in a Rat Model of Parkinson's Disease
da Silveira, S. A., Schneider, B. L., Cifuentes-Díaz, C., Sage, D., Abbas‐Terki, T., Iwatsubo, T., Unser, M. A., & Aebischer, P. (2008) · Human Molecular Genetics · doi:10.1093/hmg/ddn417 · Cited by 196
Recent developments in adeno‐associated virus vector technology
Büning, H., Perabò, L., Coutelle, O., Quadt‐Humme, S., & Hallek, M. J. (2008) · The Journal of Gene Medicine · doi:10.1002/jgm.1205 · Cited by 204
Comparison 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
The Assembly-Activating Protein Promotes Capsid Assembly of Different Adeno-Associated Virus Serotypes
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-citation
Adeno-Associated Virus Vectorology, Manufacturing, and Clinical Applications
Grieger, J. C., & Samulski, R. J. (2012) · Methods in Enzymology · doi:10.1016/b978-0-12-386509-0.00012-0 · Cited by 199
Improved TMC1 gene therapy restores hearing and balance in mice with genetic inner ear disorders
Nist-Lund, C. A., Pan, B., Patterson, A., Asai, Y., Chen, T., Zhou, W., Zhu, H., Pinto, S. R., Resnik, J., Polley, D. B., et al. (2019) · Nature Communications · doi:10.1038/s41467-018-08264-w · Cited by 183
ORANGE: A CRISPR/Cas9-based genome editing toolbox for epitope tagging of endogenous proteins in neurons
Willems, J., de Jong, A. P. H., Scheefhals, N., Mertens, E. J., Catsburg, L. A. E., Poorthuis, R. B., de Winter, F., Verhaagen, J., Meye, F. J., & MacGillavry, H. D. (2020) · PLoS Biology · doi:10.1371/journal.pbio.3000665 · Cited by 176
Critical role of RAGE and HMGB1 in inflammatory heart disease
Bangert, A., Andrassy, M., Müller, A., Bockstahler, M., Fischer, A., Volz, C. H., Leib, C., Göser, S., Korkmaz‐Icöz, S., Zittrich, S., et al. (2015) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1522288113 · Cited by 173
Silencing of Hepatic Fatty Acid Transporter Protein 5 in Vivo Reverses Diet-induced Non-alcoholic Fatty Liver Disease and Improves Hyperglycemia
Doege, H., Grimm, D., Falcon, A., Tsang, B., Storm, T. A., Xu, H., Ortegon, A. M., Kazantzis, M., Kay, M. A., & Stahl, A. (2008) · Journal of Biological Chemistry · doi:10.1074/jbc.m803510200 · Cited by 171
Self-citation
Designer Gene Delivery Vectors: Molecular Engineering and Evolution of Adeno-Associated Viral Vectors for Enhanced Gene Transfer
Kwon, I., & Schaffer, D. V. (2007) · Pharmaceutical Research · doi:10.1007/s11095-007-9431-0 · Cited by 170
Improved cardiac gene transfer by transcriptional and transductional targeting of adeno-associated viral vectors
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-citation
PLD3 affects axonal spheroids and network defects in Alzheimer’s disease
Yuan, P., Zhang, M., Tong, L., Morse, T. M., McDougal, R. A., Ding, H., Chan, D., Cai, Y., & Grutzendler, J. (2022) · Nature · doi:10.1038/s41586-022-05491-6 · Cited by 155
Split-Cre Complementation Indicates Coincident Activity of Different Genes In Vivo
Hirrlinger, J., Scheller, A., Hirrlinger, P. G., Kellert, B., Tang, W., Wehr, M. C., Goebbels, S., Reichenbach, A., Sprengel, R., Rossner, M. J., et al. (2009) · PLoS ONE · doi:10.1371/journal.pone.0004286 · Cited by 151
The Central and Basolateral Amygdala Are Critical Sites of Neuropeptide Y/Y2 Receptor-Mediated Regulation of Anxiety and Depression
Tasan, R. O., Nguyen, N. K., Weger, S., Sartori, S. B., Singewald, N., Heilbronn, R., Herzog, H., & Sperk, G. (2010) · Journal of Neuroscience · doi:10.1523/jneurosci.0430-10.2010 · Cited by 152
Analytical Ultracentrifugation as an Approach to Characterize Recombinant Adeno-Associated Viral Vectors
Burnham, B., Nass, S., Kong, E., Mattingly, M., Woodcock, D., Song, A., Wadsworth, S., Cheng, S. H., Scaria, A., & O'Riordan, C. R. (2015) · Human Gene Therapy Methods · doi:10.1089/hgtb.2015.048 · Cited by 150
Evaluation of Risks Related to the Use of Adeno-Associated Virus-Based Vectors
Tenenbaum, L., Lehtonen, E., & Monahan, P. E. (2003) · Current Gene Therapy · doi:10.2174/1566523034578131 · Cited by 146
Novel random peptide libraries displayed on AAV serotype 9 for selection of endothelial cell-directed gene transfer vectors
Varadi, K., Michelfelder, S., Korff, T., Hecker, M., Trepel, M., Katus, H. A., Kleinschmidt, J. A., & Müller, O. J. (2011) · Gene Therapy · doi:10.1038/gt.2011.143 · Cited by 147
Self-citation
Recombinant Adeno-Associated Virus Serotype 6 (rAAV2/6)-Mediated Gene Transfer to Nociceptive Neurons through Different Routes of Delivery
Towne, C. L., Pertin, M., Beggah, A., Aebischer, P., & Décosterd, I. (2009) · Molecular Pain · doi:10.1186/1744-8069-5-52 · Cited by 148
Cellular tropism and transduction properties of seven adeno-associated viral vector serotypes in adult retina after intravitreal injection
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
Silencing and un-silencing of tetracycline-controlled genes in neurons
Zhu, P., Aller, M. I., Baron, U., Cambridge, S., Bausen, M., Herb, J., Sawinski, J., Cetin, A., Osten, P., Nelson, M. L., et al. (2007) · PLoS ONE · doi:10.1371/journal.pone.0000533 · Cited by 136
Efficient optogenetic silencing of neurotransmitter release with a mosquito rhodopsin
Mahn, M., Saraf‐Sinik, I., Patil, P., Pulin, M., Bitton, E., Karalis, N., Bruentgens, F., Palgi, S., Gat, A., Dine, J., et al. (2021) · Neuron · doi:10.1016/j.neuron.2021.03.013 · Cited by 147
Adeno-Associated Virus Vectors as Therapeutic and Investigational Tools in the Cardiovascular System
Zacchigna, S., Zentilin, L., & Giacca, M. (2014) · Circulation Research · doi:10.1161/circresaha.114.302331 · Cited by 144
OneBac: Platform for Scalable and High-Titer Production of Adeno-Associated Virus Serotype 1–12 Vectors for Gene Therapy
Mietzsch, M., Grasse, S., Zurawski, C., Weger, S., Bennett, A. D., Agbandje‐McKenna, M., Muzyczka, N., Zolotukhin, S., & Heilbronn, R. (2013) · Human Gene Therapy · doi:10.1089/hum.2013.184 · Cited by 141
Driver or Coincidence Detector: Modal Switch of a Corticothalamic Giant Synapse Controlled by Spontaneous Activity and Short-Term Depression
Groh, A., de Kock, C. D., Wimmer, V., Sakmann, B., & Kuner, T. (2008) · Journal of Neuroscience · doi:10.1523/jneurosci.1554-08.2008 · Cited by 141
Radixin regulates synaptic GABAA receptor density and is essential for reversal learning and short-term memory
Hausrat, T. J., Muhia, M., Gerrow, K., Thomas, P., Hirdes, W., Tsukita, S., Heisler, F. F., Herich, L., Dubroqua, S., Breiden, P., et al. (2015) · Nature Communications · doi:10.1038/ncomms7872 · Cited by 138
Perineuronal net digestion with chondroitinase restores memory in mice with tau pathology
Yang, S., Cacquevel, M., Saksida, L. M., Bussey, T. J., Schneider, B. L., Aebischer, P., Melani, R., Pizzorusso, T., Fawcett, J. W., & Spillantini, M. G. (2015) · Experimental Neurology · doi:10.1016/j.expneurol.2014.11.013 · Cited by 138
Adeno‐associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo†‡
Paulk, N. K., Wursthorn, K., Wang, Z., Finegold, M. J., Kay, M. A., & Grompe, M. (2010) · Hepatology · doi:10.1002/hep.23481 · Cited by 135
Self-citation
Choice of template delivery mitigates the genotoxic risk and adverse impact of editing in human hematopoietic stem cells
Ferrari, S., Jacob, A., Cesana, D., Laugel, M., Beretta, S., Varesi, A., Unali, G., Conti, A., Canarutto, D., Albano, L., et al. (2022) · Cell stem cell · doi:10.1016/j.stem.2022.09.001 · Cited by 133
Virally expressed connexin26 restores gap junction function in the cochlea of conditional Gjb2 knockout mice
Yu, Q., Wang, Y., Chang, Q., Wang, J., Gong, S., Li, H., & Lin, X. (2013) · Gene Therapy · doi:10.1038/gt.2013.59 · Cited by 129
Systemic AAV6 Delivery Mediating RNA Interference Against SOD1: Neuromuscular Transduction Does Not Alter Disease Progression in fALS Mice
Towne, C. L., Raoul, C., Schneider, B. L., & Aebischer, P. (2008) · Molecular Therapy · doi:10.1038/mt.2008.73 · Cited by 137
Differential Adeno-Associated Virus Serotype-Specific Interaction Patterns with Synthetic Heparins and Other Glycans
Mietzsch, M., Broecker, F., Reinhardt, A., Seeberger, P. H., & Heilbronn, R. (2013) · Journal of Virology · doi:10.1128/jvi.03371-13 · Cited by 130
Comparison 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
Self-citation
Pathway-, layer- and cell-type-specific thalamic input to mouse barrel cortex
Sermet, B. S., Truschow, P., Feyerabend, M., Mayrhofer, J. M., Oram, T. B., Yizhar, O., Staiger, J. F., & Petersen, C. C. H. (2019) · eLife · doi:10.7554/elife.52665 · Cited by 132
Efficient transduction of non-human primate motor neurons after intramuscular delivery of recombinant AAV serotype 6
Towne, C., Schneider, B. L., Kieran, D., Redmond, D. E., & Aebischer, P. (2010) · Gene Therapy · doi:10.1038/gt.2009.119 · Cited by 124
Adeno-associated virus-mediated L1 expression promotes functional recovery after spinal cord injury
Chen, J., Wu, J., Apostolova, I. I., Skup, M., Irintchev, A., Kügler, S., & Schachner, M. (2007) · Brain · doi:10.1093/brain/awm049 · Cited by 123
The Role of the Adeno-Associated Virus Capsid in Gene Transfer
Vliet, K. V., Blouin, V., Brument, N., Agbandje‐McKenna, M., & Snyder, R. O. (2008) · Methods in molecular biology · doi:10.1007/978-1-59745-210-6_2 · Cited by 118
Conditional Single Vector CRISPR/SaCas9 Viruses for Efficient Mutagenesis in the Adult Mouse Nervous System
Hunker, A. C., Soden, M. E., Krayushkina, D., Heymann, G., Awatramani, R. B., & Zweifel, L. S. (2020) · Cell Reports · doi:10.1016/j.celrep.2020.02.092 · Cited by 124
Liver Transduction with Recombinant Adeno-Associated Virus Is Primarily Restricted by Capsid Serotype Not Vector Genotype
Grimm, 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-citation
Protection Against Henipavirus Infection by Use of Recombinant Adeno-Associated Virus–Vector Vaccines
Ploquin, A., Szécsi, J., Mathieu, C., Guillaume, V., Barateau, V., Ong, K. C., Wong, K. T., Cosset, F., Horvat, B., & Salvetti, A. (2012) · The Journal of Infectious Diseases · doi:10.1093/infdis/jis699 · Cited by 116
Effect of Adeno-Associated Virus Serotype and Genomic Structure on Liver Transduction and Biodistribution in Mice of Both Genders
Pañeda, A., Vanrell, L., Mauleon, I., Crettaz, J. S., Berraondo, P., Timmermans, E. J., Beattie, S. G., Twisk, J., van Deventer, S., Prieto, J., et al. (2009) · Human Gene Therapy · doi:10.1089/hum.2009.031 · Cited by 109
CREB-activity and nmnat2 transcription are down-regulated prior to neurodegeneration, while NMNAT2 over-expression is neuroprotective, in a mouse model of human tauopathy
Ljungberg, M. C., Ali, Y. O., Zhu, J., Wu, C.-S., Oka, K., Zhai, R. G., & Lu, H.-C. (2012) · Human Molecular Genetics · doi:10.1093/hmg/ddr492 · Cited by 107
A Molecular Basis of Analgesic Tolerance to Cannabinoids
Tappe‐Theodor, A., Agarwal, N., Katona, I., Rubino, T., Martini, L., Swiercz, J. M., Mackie, K., Monyer, H., Parolaro, D., Whistler, J. L., et al. (2007) · Journal of Neuroscience · doi:10.1523/jneurosci.5648-06.2007 · Cited by 106
Minicircle‐DNA production by site specific recombination and protein–DNA interaction chromatography
Mayrhofer, P., Blaesen, M., Schleef, M., & Jechlinger, W. (2008) · The Journal of Gene Medicine · doi:10.1002/jgm.1243 · Cited by 102
Rescue of cardiomyopathy through U7snRNA‐mediated exon skipping in Mybpc3‐targeted knock‐in mice
Gedicke, C., Behrens‐Gawlik, V., Reischmann, S., Geertz, B., Stimpel, D., Weinberger, F., Schlossarek, S., Précigout, G., Braren, I., Thomas Eschenhagen, A., et al. (2013) · EMBO Molecular Medicine · doi:10.1002/emmm.201202168 · Cited by 101
Recombinant adeno-associated virus-mediated microRNA delivery into the postnatal mouse brain reveals a role for miR-134 in dendritogenesis in vivo
Christensen, M. (2009) · Frontiers in Neural Circuits · doi:10.3389/neuro.04.016.2009 · Cited by 101
FOXO3 determines the accumulation of α-synuclein and controls the fate of dopaminergic neurons in the substantia nigra
Pino, E., Amamoto, R., Zheng, L., Cacquevel, M., Sarria, J. ‐. F., Knott, G. W., & Schneider, B. L. (2013) · Human Molecular Genetics · doi:10.1093/hmg/ddt530 · Cited by 105
Parkinson's Disease: Gene TherapiesPreprint
Coune, P., Schneider, B. L., & Aebischer, P. (2012) · Cold Spring Harbor Perspectives in Medicine · doi:10.1101/cshperspect.a009431 · Cited by 102
Viral Vectors for Gene Transfer: Current Status of Gene Therapeutics
Heilbronn, R., & Weger, S. (2009) · Handbook of experimental pharmacology · doi:10.1007/978-3-642-00477-3_5 · Cited by 98
Gene therapy in the cornea
Mohan, R. R., Sharma, A., Netto, M. V., Sinha, S., & Wilson, S. E. (2005) · Progress in Retinal and Eye Research · doi:10.1016/j.preteyeres.2005.04.001 · 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-citation
AAVs Anatomy: Roadmap for Optimizing Vectors for Translational Success
Mitchell, A. M., Nicolson, S. C., Warischalk, J. K., & Samulski, R. J. (2010) · Current Gene Therapy · doi:10.2174/156652310793180706 · Cited by 97
Syntaxin 5-Dependent Retrograde Transport to the trans -Golgi Network Is Required for Adeno-Associated Virus Transduction
Nonnenmacher, M. E., Cintrat, J., Gillet, D., & Weber, T. (2014) · Journal of Virology · doi:10.1128/jvi.02520-14 · Cited by 96
Cardio-specific long-term gene expression in a porcine model after selective pressure-regulated retroinfusion of adeno-associated viral (AAV) vectors
Raake, P. W. J., Hinkel, R., Müller, S., Delker, S., Kreuzpointner, R., Kupatt, C., Katus, H. A., Kleinschmidt, J. A., Boekstegers, P., & Müller, O. J. (2007) · Gene Therapy · doi:10.1038/sj.gt.3303035 · Cited by 93
Self-citation
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
Self-citation
Gene Therapy Strategies for Duchenne Muscular Dystrophy Utilizing Recombinant Adeno-associated Virus Vectors
Blankinship, M. J., Gregorevic, P., & Chamberlain, J. S. (2005) · Molecular Therapy · doi:10.1016/j.ymthe.2005.11.001 · Cited by 93
Manufacturing 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
Adeno-associated Virus Vector Serotypes Mediate Sustained Correction of Bilirubin UDP Glucuronosyltransferase Deficiency in Rats
Seppen, J., Bakker, C., de Jong, B., Kunne, C., van den Oever, K., Vandenberghe, K., de Waart, R. D., Twisk, J., & Bosma, P. J. (2006) · Molecular Therapy · doi:10.1016/j.ymthe.2006.01.014 · Cited by 87
Cellular toxicity following application of adeno-associated viral vector-mediated RNA interference in the nervous system
Ehlert, E., Eggers, R., Niclou, S. P., & Verhaagen, J. (2010) · BMC Neuroscience · doi:10.1186/1471-2202-11-20 · Cited by 85
A Two-hybrid Screen Identifies Cathepsins B and L as Uncoating Factors for Adeno-associated Virus 2 and 8
Akache, B., Grimm, D., Shen, X., Fuess, S., Yant, S. R., Glazer, D. S., Park, J., & Kay, M. A. (2007) · Molecular Therapy · doi:10.1038/sj.mt.6300053 · Cited by 87
Self-citation
Understanding Capsid Assembly and Genome Packaging for Adeno-Associated Viruses
Bennett, A., Mietzsch, M., & Agbandje-McKenna, M. (2017) · Future Virology · doi:10.2217/fvl-2017-0011 · Cited by 82
Recombinant Adeno-Associated Virus As Delivery Vector for Gene Therapy—A Review
Yang, L. (2004) · Stem Cells and Development · doi:10.1089/154732804773099335 · Cited by 81
Correction of Feline Lipoprotein Lipase Deficiency with Adeno-Associated Virus Serotype 1-Mediated Gene Transfer of the Lipoprotein Lipase S447X Beneficial Mutation
Ross, C. J., Twisk, J., Bakker, A. C., Miao, F., Verbart, D. J., Rip, J., Godbey, T., Dijkhuizen, P. A., Hermens, W. T. J. M. C., Kastelein, J. J. P., et al. (2006) · Human Gene Therapy · doi:10.1089/hum.2006.17.487 · Cited by 82
Protocol for Efficient Generation and Characterization of Adeno-Associated Viral Vectors
Jungmann, A., Leuchs, B., Rommelaere, J., Katus, H. A., & Müller, O. J. (2017) · Human Gene Therapy Methods · doi:10.1089/hgtb.2017.192 · Cited by 79
Decreased SGK1 Expression and Function Contributes to Behavioral Deficits Induced by Traumatic Stress
Licznerski, P., Duric, V., Banasr, M., Alavian, K. N., Ota, K. T., Kang, H. J., Jonas, E. A., Ursano, R., Krystal, J. H., Duman, R. S., et al. (2015) · PLOS Biology · doi:10.1371/journal.pbio.1002282 · Cited by 79
Long-Term Correction of Murine Lipoprotein Lipase Deficiency with AAV1-Mediated Gene Transfer of the Naturally Occurring LPL S447X Beneficial Mutation
Ross, C. J., Twisk, J., Meulenberg, J. J. M., Liu, G., van den Oever, K., Moraal, E., Hermens, W. T., Rip, J., Kastelein, J. J. P., Kuivenhoven, J. A., et al. (2004) · Human Gene Therapy · doi:10.1089/hum.2004.15.906 · Cited by 78
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-citation
Rapid characterization of adeno-associated virus (AAV) gene therapy vectors by mass photometry
Wu, D., Hwang, P., Li, T., & Piszczek, G. (2022) · Gene Therapy · doi:10.1038/s41434-021-00311-4 · Cited by 83
Interference with the production of infectious viral particles and bimodal inhibition of replication are broadly conserved antiviral properties of IFITMs
Tartour, K., Nguyen, X.-N., Appourchaux, R., Assil, S., Barateau, V., Bloyet, L.-M., Burlaud Gaillard, J., Confort, M.-P., Escudero-Perez, B., Gruffat, H., et al. (2017) · PLOS Pathogens · doi:10.1371/journal.ppat.1006610 · Cited by 69
3D super-resolution deep-tissue imaging in living mice
Velasco, M. G. M., Zhang, M., Antonello, J., Yuan, P., Allgeyer, E. S., May, D., M’Saad, O., Kidd, P., Barentine, A. E. S., Greco, V., et al. (2021) · Optica · doi:10.1364/optica.416841 · Cited by 77
Recombinant adeno-associated viral (rAAV) vectors as therapeutic tools for Duchenne muscular dystrophy (DMD)
Athanasopoulos, T., Graham, I., Foster, H., & Dickson, G. (2004) · Gene Therapy · doi:10.1038/sj.gt.3302379 · Cited by 82
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-citation
Repair of Mybpc3 mRNA by 5′-trans-splicing in a Mouse Model of Hypertrophic Cardiomyopathy
Mearini, G., Stimpel, D., Krämer, E., Geertz, B., Braren, I., Gedicke, C., Précigout, G., Müller, O. J., Katus, H. A., Thomas Eschenhagen, A., et al. (2013) · Molecular Therapy — Nucleic Acids · doi:10.1038/mtna.2013.31 · Cited by 74
Hypothalamic glycogen synthase kinase 3β has a central role in the regulation of food intake and glucose metabolism
Benzler, J., Ganjam, G. K., Krüger, M., Pinkenburg, O., Kutschke, M., Stöhr, S., Steger, J., Koch, C. E., Oelkrug, R., Schwartz, M. W., et al. (2012) · Biochemical Journal · doi:10.1042/bj20120834 · Cited by 70
RNA interference: Implications for cancer treatment
Masiero, M., Nardo, G., Indraccolo, S., & Favaro, E. (2007) · Molecular Aspects of Medicine · doi:10.1016/j.mam.2006.12.004 · Cited by 71
Sources of off-target expression from recombinase-dependent AAV vectors and mitigation with cross-over insensitive ATG-out vectors
Fischer, K. B., Collins, H. K., & Callaway, E. M. (2019) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1915974116 · Cited by 69
Modulation of feeding by chronic rAAV expression of a relaxin-3 peptide agonist in rat hypothalamus
Ganella, D. E., Callander, G. E., Ma, S., Bye, C. R., Gundlach, A. L., & Bathgate, R. A. D. (2012) · Gene Therapy · doi:10.1038/gt.2012.83 · Cited by 70
Heart-targeted adeno-associated viral vectors selected by in vivo biopanning of a random viral display peptide library
Ying, Y., Müller, O. J., Goehringer, C., Leuchs, B., Trepel, M., Katus, H. A., & Kleinschmidt, J. A. (2010) · Gene Therapy · doi:10.1038/gt.2010.44 · Cited by 69
Self-citation
AAV2 production with optimized N/P ratio and PEI-mediated transfection results in low toxicity and high titer for in vitro and in vivo applications
Huang, X., Hartley, A.-V., Yin, Y., Herskowitz, J. H., Lah, J. J., & Ressler, K. J. (2013) · Journal of Virological Methods · doi:10.1016/j.jviromet.2013.06.008 · Cited by 68
Synthetic riboswitches for external regulation of genes transferred by replication-deficient and oncolytic adenoviruses
Ketzer, P., Haas, S. F., Engelhardt, S., Hartig, J. S., & Nettelbeck, D. M. (2012) · Nucleic Acids Research · doi:10.1093/nar/gks734 · Cited by 67

Authors: Grimm, D., Kay, M. A., & Kleinschmidt, J. A.
Affiliations: Stanford Medicine (US)
German Cancer Research Center (DE)
Corresponding author: Kay, Mark A
ORCID: Grimm, Dirk
Kay, Mark A
Venue: Molecular Therapy
Publisher: Elsevier BV
Published: 2003-06
Volume: 7
Issue: 6
Pages: 839-850
ISSN: 1525-0016 (ISSN-L)
DOI: 10.1016/s1525-0016(03)00095-9
OpenAlex: W1977797303
Cited by: 344
References: 36
Citations by year: 2026: 4 · 2025: 9 · 2024: 12 · 2023: 10 · 2022: 15 · 2021: 17 · 2020: 12 · 2019: 18 · 2018: 5 · 2017: 17 · 2016: 7 · 2015: 21 · 2014: 12 · 2013: 20 · 2012: 19
FWCI: 7.96
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, 4877 d)
Locations: Molecular Therapy (publishedVersion, cc-by-nc-nd)
PubMed (publishedVersion)
Funders: National Heart, Lung, and Blood Institute (HL66948, U01HL066948)
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 vectors, fluorescent protein markers, cell tropism, transgene expression kinetics, gene therapy vectors
MeSH terms: Dependovirus, Cell Line, DNA Replication, DNA-Binding Proteins, Genetic Vectors, Helper Viruses, Humans, Plasmids, Serotyping, Viral Proteins, Virus Replication, Virus Assembly

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

Helper virus-free, optically controllable, and two-plasmid-based production of adeno-associated virus vectors of serotypes 1 to 6

Abstract

Overview

Integrity signals

Verification

Claims the paper makes

Claims other papers make about this one

Self-check

Citations

Cites

Cited by

Related

Metadata

Record

About these checks