The DOI matches its Crossref record; 48 of the 49 references it cites were found in a registry or an index.
Engineering and Selection of Shuffled AAV Genomes: A New Strategy for Producing Targeted Biological Nanoparticles
Open accessAbstract
We report a DNA shuffling-based approach for developing cell type-specific vectors through directed evolution. Capsid genomes of adeno-associated virus (AAV) serotypes 1-9 were randomly fragmented and reassembled using PCR to generate a chimeric capsid library. A single infectious clone (chimeric-1829) containing genome fragments from AAV1, 2, 8, and 9 was isolated from an integrin minus hamster melanoma cell line previously shown to have low permissiveness to AAV. Molecular modeling studies suggest that AAV2 contributes to surface loops at the icosahedral threefold axis of symmetry, while AAV1 and 9 contribute to two- and fivefold symmetry interactions, respectively. The C-terminal domain (AAV9) was identified as a critical structural determinant of melanoma tropism through rational mutagenesis. Chimeric-1829 utilizes heparan sulfate as a primary receptor and transduces melanoma cells more efficiently than all serotypes. Further, chimeric-1829 demonstrates altered tropism in rodent skeletal muscle, liver, and brain including nonhuman primates. We determined a unique immunological profile based on neutralizing antibody (NAb) titer and crossreactivity studies strongly supporting isolation of a synthetic laboratory-derived capsid variant. Application of this technology to alternative cell/tissue types using AAV or other viral capsid sequences is likely to yield a new class of biological nanoparticles as vectors for human gene transfer.
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
“A novel variant selected from this screen-chimeric-1829-showed higher transduction efficiency and low cross-reactivity with the pre-existing antibodies against other serotypes, indicating that the immune response may not recognize chimeric-1829 [90].”
From Immune responses to gene editing by viral and non-viral delivery vectors used in retinal gene therapy (2022) · cites this paper as [90]
Citations
Cites
Jones, T. A., Zou, J.-Y., Cowan, S. W., & Kjeldgaard, M. (1991) · Acta Crystallographica Section A Foundations of Crystallography · doi:10.1107/s0108767390010224 · Cited by 12,616
SWISS-MODEL: an automated protein homology-modeling server
Schwede, T. F. (2003) · Nucleic Acids Research · doi:10.1093/nar/gkg520 · Cited by 5,592
Rapid evolution of a protein in vitro by DNA shuffling
Stemmer, W. P. C. (1994) · Nature · doi:10.1038/370389a0 · Cited by 1,932
Summerford, C., & Samulski, R. J. (1998) · Journal of Virology · doi:10.1128/jvi.72.2.1438-1445.1998 · Cited by 1,409
Self-citationXiao, X., Li, J., & Samulski, R. J. (1998) · Journal of Virology · doi:10.1128/jvi.72.3.2224-2232.1998 · Cited by 1,381
Self-citationImproved Green Fluorescent Protein by Molecular Evolution Using DNA Shuffling
Crameri, A., Whitehorn, E. A., Tate, E. H., & Stemmer, W. P. C. (1996) · Nature Biotechnology · doi:10.1038/nbt0396-315 · Cited by 1,187
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
Self-citationDNA shuffling of a family of genes from diverse species accelerates directed evolution
Crameri, A., Raillard, S. A., Bermudez, E., & Stemmer, W. P. C. (1998) · Nature · doi:10.1038/34663 · Cited by 826
Jones, N. C., & Shenk, T. E. (1979) · Cell · doi:10.1016/0092-8674(79)90275-7 · Cited by 784
Unraveling hot spots in binding interfaces: progress and challenges
DeLano, W. L. (2002) · Current Opinion in Structural Biology · doi:10.1016/s0959-440x(02)00283-x · Cited by 767
Flagged first, then by citations
1 of the 49 references couldn’t be matched to a known paper.
Cited by
Sorted by citations
Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes
Foust, K. D., Nurre, E., Montgomery, C. L., Hernandez, A., Chan, C. M., & Kaspar, B. K. (2009) · Nature Biotechnology · doi:10.1038/nbt.1515 · Cited by 1,456
Adeno-associated virus as a delivery vector for gene therapy of human diseases
Wang, J.-H., Gessler, D. J., Zhan, W., Gallagher, T. L., & Gao, G. (2024) · Signal Transduction and Targeted Therapy · doi:10.1038/s41392-024-01780-w · Cited by 904
Engineering adeno-associated viruses for clinical gene therapy
Kotterman, M. A., & Schaffer, D. V. (2014) · Nature Reviews Genetics · doi:10.1038/nrg3742 · Cited by 761
State-of-the-art gene-based therapies: the road ahead
Kay, M. A. (2011) · Nature Reviews Genetics · doi:10.1038/nrg2971 · Cited by 698
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-citationTherapeutic in vivo delivery of gene editing agents
Raguram, A., Banskota, S., & Liu, D. R. (2022) · Cell · doi:10.1016/j.cell.2022.03.045 · Cited by 552
Gray, S. J., Matagne, V., Bachaboina, L., Yadav, S., Ojeda, S. R., & Samulski, R. J. (2011) · Molecular Therapy · doi:10.1038/mt.2011.72 · Cited by 501
Self-citationSelection and evaluation of clinically relevant AAV variants in a xenograft liver model
Lisowski, L., Dane, A. P., Chu, K., Zhang, Y., Cunningham, S. C., Wilson, E. M., Nygaard, S., Grompe, M., Alexander, I. E., & Kay, M. A. (2014) · Nature · doi:10.1038/nature12875 · Cited by 450
Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector
Bowles, D. E., McPhee, S. W., Li, C., Gray, S. J., Samulski, J. J., Camp, A. S., Li, J., Wang, B., Monahan, P. E., Rabinowitz, J. E., et al. (2012) · Molecular Therapy · doi:10.1038/mt.2011.237 · Cited by 400
Self-citationAAV vectors: The Rubik’s cube of human gene therapy
Pupo, A., Fernández, A., Low, S. H., François, A., Suárez-Amarán, L., & Samulski, R. J. (2022) · Molecular Therapy · doi:10.1016/j.ymthe.2022.09.015 · Cited by 382
Self-citationKeeler, A. M., & Flotte, T. R. (2019) · Annual Review of Virology · doi:10.1146/annurev-virology-092818-015530 · Cited by 322
In Silico Reconstruction of the Viral Evolutionary Lineage Yields a Potent Gene Therapy Vector
Zinn, E., Pacouret, S., Khaychuk, V., Turunen, H. T., Carvalho, L. S., Andres-Mateos, E., Shah, S., Shelke, R., Maurer, A. C., Plovie, E., et al. (2015) · Cell Reports · doi:10.1016/j.celrep.2015.07.019 · Cited by 310
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
Nathanson, J. L., Yanagawa, Y., Obata, K., & Callaway, E. M. (2009) · Neuroscience · doi:10.1016/j.neuroscience.2009.03.032 · Cited by 305
Zhang, H., Yang, B., Mu, X., Ahmed, S. S., Su, Q., He, R., Wang, H., Mueller, C., Sena-Esteves, M., Brown, R., et al. (2011) · Molecular Therapy · doi:10.1038/mt.2011.98 · Cited by 291
Ellis, B. L., Hirsch, M. L., Barker, J. C., Connelly, J. P., Steininger, R. J., & Porteus, M. H. (2013) · Virology Journal · doi:10.1186/1743-422x-10-74 · Cited by 252
Bucher, K., Rodríguez-Bocanegra, E., Dauletbekov, D., & Fischer, M. D. (2021) · Progress in Retinal and Eye Research · doi:10.1016/j.preteyeres.2020.100915 · Cited by 242
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
Adeno-associated virus (AAV) vectors in cancer gene therapy
Santiago-Ortiz, J. L., & Schaffer, D. V. (2016) · Journal of Controlled Release · doi:10.1016/j.jconrel.2016.01.001 · Cited by 243
Structural insight into the unique properties of adeno-associated virus serotype 9
DiMattia, M. A., Nam, H.-J., Van Vliet, K., Mitchell, M., Bennett, A., Gurda, B. L., McKenna, R., Olson, N. H., Sinkovits, R. S., Potter, M., et al. (2012) · Journal of Virology · doi:10.1128/jvi.07232-11 · Cited by 219
Self-citationLi, C., Narkbunnam, N., Samulski, R. J., Asokan, A., Hu, G., Jacobson, L. J., Manco-Johnson, M. J., Monahan, P. E., & The Joint Outcome Study Investigators (2012) · Gene Therapy · doi:10.1038/gt.2011.90 · Cited by 225
Self-citationRecombination in Eukaryotic Single Stranded DNA Viruses
Martin, D. P., Biagini, P., Lefeuvre, P., Golden, M., Roumagnac, P., & Varsani, A. (2011) · Viruses · doi:10.3390/v3091699 · Cited by 232
A myocardium tropic adeno-associated virus (AAV) evolved by DNA shuffling and in vivo selection
Yang, L., Jiang, J., Drouin, L. M., Agbandje‐McKenna, M., Chen, C., Qiao, C., Pu, D., Hu, X., Wang, D.-Z., Li, J., et al. (2009) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0813207106 · Cited by 213
Self-citationAn AAV capsid reprogrammed to bind human transferrin receptor mediates brain-wide gene delivery
Huang, Q., Chan, K. Y., Wu, J. H., Botticello-Romero, N. R., Zheng, Q., Lou, S., Keyes, C., Svanbergsson, A., Johnston, J., Mills, A., et al. (2024) · Science · doi:10.1126/science.adm8386 · Cited by 201
Sarcoplasmic reticulum Ca2+ATPase as a therapeutic target for heart failure
Lipskaia, L., Chemaly, E. R., Hadri, L., Lompré, A., & Hajjar, R. J. (2009) · Expert Opinion on Biological Therapy · doi:10.1517/14712590903321462 · Cited by 201
Molecular evolution of adeno-associated virus for enhanced glial gene delivery
Koerber, J. T., Klimczak, R., Jang, J.-H., Dalkara, D., Flannery, J. G., & Schaffer, D. V. (2009) · Molecular Therapy · doi:10.1038/mt.2009.184 · Cited by 181
Gray, S. J., Blake, B. L., Criswell, H. E., Nicolson, S. C., Samulski, R. J., & McCown, T. J. (2010) · Molecular Therapy · doi:10.1038/mt.2009.292 · Cited by 185
Self-citationDNA Shuffling of Adeno-associated Virus Yields Functionally Diverse Viral Progeny
Koerber, J. T., Jang, J.-H., & Schaffer, D. V. (2008) · Molecular Therapy · doi:10.1038/mt.2008.167 · Cited by 181
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
Self-citationDirected evolution of adeno-associated virus to an infectious respiratory virus
Excoffon, K. J. D. A., Koerber, J. T., Dickey, D. D., Murtha, M. J., Keshavjee, S. H., Kaspar, B. K., Zabner, J., & Schaffer, D. V. (2009) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0813365106 · Cited by 178
Protein Cages: From Fundamentals to Advanced Applications
Edwardson, T. G. W., Levasseur, M. D., Tetter, S., Steinauer, A., Hori, M., & Hilvert, D. (2022) · Chemical Reviews · doi:10.1021/acs.chemrev.1c00877 · Cited by 171
Tailoring the AAV vector capsid for gene therapy
Vandenberghe, L. H., Wilson, J. M., & Gao, G. (2009) · Gene Therapy · doi:10.1038/gt.2008.170 · Cited by 155
Directed evolution of novel adeno-associated viruses for therapeutic gene delivery
Bartel, M. A., Weinstein, J. R., & Schaffer, D. V. (2012) · Gene Therapy · doi:10.1038/gt.2012.20 · 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
Efficient recovery of dysferlin deficiency by dual adeno-associated vector-mediated gene transfer
Lostal, W., Bartoli, M., Bourg, N., Roudaut, C., Bentaïb, A., Miyake, K., Guerchet, N., Fougerousse, F., McNeil, P., & Richard, I. (2010) · Human Molecular Genetics · doi:10.1093/hmg/ddq065 · Cited by 173
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
“In addition to peptide insertion, the development of capsid shuffling [93] and directed evolution [94], discussed in Chapter 11, has generated many promising novel AAV variants.”
BackgroundAdeno-Associated Virus Toolkit to Target Diverse Brain Cells
Challis, R. C., Ravindra Kumar, S., Chen, X., Goertsen, D., Coughlin, G. M., Hori, A. M., Chuapoco, M. R., Otis, T. S., Miles, T. F., & Gradinaru, V. (2022) · Annual Review of Neuroscience · doi:10.1146/annurev-neuro-111020-100834 · Cited by 148
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
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 144
Davidsson, M., Wang, G., Aldrin-Kirk, P., Cardoso, T., Nolbrant, S., Hartnor, M., Mudannayake, J., Parmar, M., & Björklund, T. (2019) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1910061116 · Cited by 142
Tseng, Y.-S., & Agbandje‐McKenna, M. (2014) · Frontiers in Immunology · doi:10.3389/fimmu.2014.00009 · Cited by 143
Self-citationAdeno-Associated Virus (AAV) Versus Immune Response
Rabinowitz, J., Chan, Y. K., & Samulski, R. J. (2019) · Viruses · doi:10.3390/v11020102 · Cited by 122
Self-citationAdeno-associated virus: fit to serve
Zinn, E. M., & Vandenberghe, L. H. (2014) · Current Opinion in Virology · doi:10.1016/j.coviro.2014.07.008 · Cited by 123
Gene Therapy: Design and Prospects for Craniofacial Regeneration
Scheller, E. L., & Krebsbach, P. (2009) · Journal of Dental Research · doi:10.1177/0022034509337480 · Cited by 132
Engineering Delivery Vehicles for Genome Editing
Nelson, C. E., & Gersbach, C. A. (2016) · Annual Review of Chemical and Biomolecular Engineering · doi:10.1146/annurev-chembioeng-080615-034711 · Cited by 120
Adeno-Associated Virus Structural Biology as a Tool in Vector Development
Drouin, L. M., & Agbandje-McKenna, M. (2013) · Future Virology · doi:10.2217/fvl.13.112 · Cited by 114
Self-citationIn Vivo Selection Yields AAV-B1 Capsid for Central Nervous System and Muscle Gene Therapy
Choudhury, S., Fitzpatrick, Z., Harris, A. F., Maitland, S. A., Ferreira, J. S., Zhang, Y., Ma, S., Sharma, R. B., Gray‐Edwards, H. L., Johnson, J. A., et al. (2016) · Molecular Therapy · doi:10.1038/mt.2016.84 · Cited by 118
Self-citationEngineering the AAV capsid to optimize vector–host-interactions
Büning, H., Huber, A., Zhang, L. N., Meumann, N., & Hacker, U. T. (2015) · Current Opinion in Pharmacology · doi:10.1016/j.coph.2015.08.002 · Cited by 119
Grimm, D., & Zolotukhin, S. (2015) · Molecular Therapy · doi:10.1038/mt.2015.173 · Cited by 108
Wang, L., Bell, P. L., Somanathan, S., Wang, Q., He, Z., Yu, H., McMenamin, D., Goode, T. L., Calcedo, R., & Wilson, J. M. (2015) · Molecular Therapy · doi:10.1038/mt.2015.179 · Cited by 106
A Precise Chemical Strategy To Alter the Receptor Specificity of the Adeno‐Associated Virus
Kelemen, R. E., Mukherjee, R., Cao, X., Erickson, S. B., Zheng, Y., & Chatterjee, A. (2016) · Angewandte Chemie International Edition · doi:10.1002/anie.201604067 · Cited by 98
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
RNA Encapsidation by SV40-Derived Nanoparticles Follows a Rapid Two-State Mechanism
Kler, S., Asor, R., Li, C., Ginsburg, A., Harries, D., Oppenheim, A., Zlotnick, A., & Raviv, U. (2012) · Journal of the American Chemical Society · doi:10.1021/ja2110703 · Cited by 98
Structural insights into adeno-associated virus serotype 5
Govindasamy, L., DiMattia, M. A., Gurda, B. L., Halder, S., McKenna, R., Chiorini, J. A., Muzyczka, N., Zolotukhin, S., & Agbandje-McKenna, M. (2013) · Journal of Virology · doi:10.1128/jvi.00867-13 · Cited by 95
Self-citationProduction, 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
Li, C., Diprimio, N., Bowles, D. E., Hirsch, M. L., Monahan, P. E., Asokan, A., Rabinowitz, J., Agbandje-McKenna, M., & Samulski, R. J. (2012) · Journal of Virology · doi:10.1128/jvi.00675-12 · Cited by 96
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 99
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
Self-citationUsing a barcoded AAV capsid library to select for clinically relevant gene therapy vectors
Pekrun, K., de Alencastro, G., Luo, Q.-J., Liu, J., Kim, Y., Nygaard, S. C., Galivo, F., Zhang, F., Song, R., Tiffany, M. R., et al. (2019) · JCI Insight · doi:10.1172/jci.insight.131610 · Cited by 98
AAV9: a potential blood-brain barrier buster
Manfredsson, F. P., Rising, A. C., & Mandel, R. J. (2009) · Molecular Therapy · doi:10.1038/mt.2009.15 · Cited by 94
Characterization of a novel adeno-associated viral vector with preferential oligodendrocyte tropism
Powell, S. K., Khan, N., Parker, C. L., Samulski, R. J., Matsushima, G., Gray, S. J., & McCown, T. J. (2016) · Gene Therapy · doi:10.1038/gt.2016.62 · Cited by 92
Self-citationStructure of AAV-DJ, a Retargeted Gene Therapy Vector: Cryo-Electron Microscopy at 4.5 Å Resolution
Lerch, T. F., O’Donnell, J., Meyer, N., Xie, Q., Taylor, K. A., Stagg, S. M., & Chapman, M. S. (2012) · Structure · doi:10.1016/j.str.2012.05.004 · Cited by 88
Li, Y., Zeng, H., Xu, R., Liu, B., & Li, Z. (2009) · Stem Cells · doi:10.1002/stem.234 · Cited by 85
Li, W., Zhang, L., Johnson, J. S., Wu, Z., Grieger, J. C., Xiao, P., Drouin, L. M., Agbandje‐McKenna, M., Pickles, R. J., & Samulski, R. J. (2009) · Molecular Therapy · doi:10.1038/mt.2009.155 · Cited by 89
Self-citationAdeno-associated Virus as a Mammalian DNA Vector
Salganik, M., Hirsch, M. L., & Samulski, R. J. (2015) · Microbiology Spectrum · doi:10.1128/microbiolspec.mdna3-0052-2014 · Cited by 93
Self-citationState-of-the-art human gene therapy: part I. Gene delivery technologies.
Wang, D., & Gao, G. (2014) · PubMed · Cited by 85
Little Vector, Big Gene Transduction: Fragmented Genome Reassembly of Adeno-associated Virus
Hirsch, M. L., Agbandje‐McKenna, M., & Samulski, R. J. (2010) · Molecular Therapy · doi:10.1038/mt.2009.280 · Cited by 86
Self-citationCRISPR genome engineering and viral gene delivery: A case of mutual attraction
Schmidt, F., & Grimm, D. (2015) · Biotechnology Journal · doi:10.1002/biot.201400529 · Cited by 85
Therapeutic applications of genetic code expansion
Huang, Y., & Liu, T. (2018) · Synthetic and Systems Biotechnology · doi:10.1016/j.synbio.2018.09.003 · Cited by 82
Becker, J., Fakhiri, J., & Grimm, D. (2022) · Pathogens · doi:10.3390/pathogens11070756 · Cited by 88
Ferret and Pig Models of Cystic Fibrosis: Prospects and Promise for Gene Therapy
Yan, Z., Stewart, Z. A., Sinn, P. L., Olsen, J. C., Hu, J., McCray, P. B., & Engelhardt, J. F. (2014) · Human Gene Therapy Clinical Development · doi:10.1089/humc.2014.154 · Cited by 81
Chowdhury, E. A., Meno‐Tetang, G. M. L., Chang, H. Y., Wu, S., Huang, H. W., Jamier, T., Chandran, J. S., & Shah, D. K. (2021) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2021.01.017 · Cited by 80
Michelfelder, 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
Marsic, D., Govindasamy, L., Currlin, S., Markusic, D. M., Tseng, Y.-S., Herzog, R. W., Agbandje‐McKenna, M., & Zolotukhin, S. (2014) · Molecular Therapy · doi:10.1038/mt.2014.139 · Cited by 75
Self-citationGenome engineering: a new approach to gene therapy for neuromuscular disorders
Nelson, C. E., Robinson-Hamm, J. N., & Gersbach, C. A. (2017) · Nature Reviews Neurology · doi:10.1038/nrneurol.2017.126 · Cited by 73
The next step in gene delivery: Molecular engineering of adeno-associated virus serotypes
Wang, J., Faust, S. M., & Rabinowitz, J. E. (2010) · Journal of Molecular and Cellular Cardiology · doi:10.1016/j.yjmcc.2010.10.017 · Cited by 69
Srivastava, A. (2023) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2023.05.014 · Cited by 68
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
Structure of neurotropic adeno-associated virus AAVrh.8
Halder, S., Vliet, K. V., Smith, J. K., Duong, T. T. P., McKENNA, R. V., Wilson, J. M., & Agbandje‐McKenna, M. (2015) · Journal of Structural Biology · doi:10.1016/j.jsb.2015.08.017 · Cited by 62
Self-citationAdeno-Associated Virus Biology
Weitzman, M. D., & Linden, R. M. (2012) · Methods in Molecular Biology · doi:10.1007/978-1-61779-370-7_1 · Cited by 132
Butler, D. C., McLear, J. A., & Messer, A. C. (2011) · Progress in Neurobiology · doi:10.1016/j.pneurobio.2011.11.004 · Cited by 63
Carvalho, M. A. B. R., Sepodes, B. M. N., & Martins, A. P. (2017) · Frontiers in Medicine · doi:10.3389/fmed.2017.00182 · Cited by 65
Viral vectors as vaccine carriers
Ertl, H. C. J. (2016) · Current Opinion in Virology · doi:10.1016/j.coviro.2016.06.001 · Cited by 62
Große, S., Penaud‐Budloo, M., Herrmann, A.-K., Börner, K., Fakhiri, J., Laketa, V., Krämer, C., Wiedtke, E., Gunkel, M., Ménard, L., et al. (2017) · Journal of Virology · doi:10.1128/jvi.01198-17 · Cited by 62
A versatile toolkit for overcoming AAV immunity
Li, X., Wei, X., Lin, J., & Ou, L. (2022) · Frontiers in Immunology · doi:10.3389/fimmu.2022.991832 · Cited by 57
Galibert, L., & Merten, O. (2011) · Journal of Invertebrate Pathology · doi:10.1016/j.jip.2011.05.008 · Cited by 61
Applying machine learning to predict viral assembly for adeno-associated virus capsid libraries
Marques, A. D., Kummer, M., Kondratov, O., Banerjee, A., Moskalenko, O. V., & Zolotukhin, S. (2020) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2020.11.017 · Cited by 55
Gene delivery methods in cardiac gene therapy
Ishikawa, K., Tilemann, L. M., Fish, K. M., & Hajjar, R. J. (2011) · The Journal of Gene Medicine · doi:10.1002/jgm.1609 · Cited by 55
Adeno-Associated Virus (AAV) Gene Delivery: Dissecting Molecular Interactions upon Cell Entry
Large, E. E., Silveria, M., Zane, G. M., Weerakoon, O., & Chapman, M. S. (2021) · Viruses · doi:10.3390/v13071336 · Cited by 57
Sugano, E., Isago, H., Wang, Z., Murayama, N., Tamai, M., & Tomita, H. (2010) · Gene Therapy · doi:10.1038/gt.2010.140 · Cited by 55
Synthetic virology: engineering viruses for gene delivery
Guenther, C. M., Kuypers, B. E., Lam, M. T., Robinson, T. M., Zhao, J. X., & Suh, J. (2014) · Wiley Interdisciplinary Reviews Nanomedicine and Nanobiotechnology · doi:10.1002/wnan.1287 · Cited by 56
Next-generation AAV vectors for clinical use: an ever-accelerating race
Weinmann, J., & Grimm, D. (2017) · Virus Genes · doi:10.1007/s11262-017-1502-7 · Cited by 54
Ren, D., Fisson, S., Dalkara, D., & Ail, D. (2022) · Pharmaceutics · doi:10.3390/pharmaceutics14091973 · Cited by 52
Zhou, L., Ni, Y.-Y., Piñeyro, P. E., Sanford, B. J., Cossaboom, C. M., Dryman, B. A., Huang, Y., Cao, D., & Meng, X. (2012) · Virology · doi:10.1016/j.virol.2012.09.005 · Cited by 52
Optimization of AAV vectors to target persistent viral reservoirs
Colón-Thillet, R., Jerome, K. R., & Stone, D. (2021) · Virology Journal · doi:10.1186/s12985-021-01555-7 · Cited by 50
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
Nance, M. E., & Duan, D. (2015) · Human Gene Therapy · doi:10.1089/hum.2015.107 · Cited by 52
Judd, J., Wei, F., Nguyen, P. Q., Tartaglia, L. J., Agbandje‐McKenna, M., Silberg, J. J., & Suh, J. (2012) · Molecular Therapy — Nucleic Acids · doi:10.1038/mtna.2012.46 · Cited by 53
Self-citationDirected evolution of adeno-associated virus for glioma cell transduction
Maguire, C. A., Gianni, D., Meijer, D. H., Shaket, L. A., Wakimoto, H., Rabkin, S. D., Gao, G., & Sena‐Esteves, M. (2009) · Journal of Neuro-Oncology · doi:10.1007/s11060-009-9972-7 · Cited by 52
CAV-2 Vector Development and Gene Transfer in the Central and Peripheral Nervous Systems
Rio, D. D., Beucher, B., Lavigne, M., Wehbi, A., Dopeso‐Reyes, I. G., Saggio, I., & Kremer, E. J. (2019) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2019.00071 · Cited by 53
Metadata
- Authors
- Li, W., Asokan, A., Wu, Z., Van Dyke, T., DiPrimio, N., Johnson, J. S., Govindaswamy, L., Agbandje-McKenna, M., Leichtle, S., Eugene Redmond Jr, D., McCown, T. J., Petermann, K. B., Sharpless, N. E., & Samulski, R. J.
- Affiliations
- University of North Carolina at Chapel Hill (US)
- University of Florida (US)
- Yale University (US)
- Corresponding author
- Samulski, Richard J
- ORCID
- Venue
- Molecular Therapy
- Publisher
- Elsevier BV
- Published
- 2008-07
- Volume
- 16
- Issue
- 7
- Pages
- 1252-1260
- ISSN
- 1525-0016 (ISSN-L)
- PMC
- PMC2632803
- OpenAlex
- W2113082692
- Cited by
- 261
- References
- 49
- Citations by year
- 2026: 8 · 2025: 8 · 2024: 13 · 2023: 6 · 2022: 16 · 2021: 11 · 2020: 10 · 2019: 15 · 2018: 12 · 2017: 14 · 2016: 9 · 2015: 15 · 2014: 16 · 2013: 14 · 2012: 24
- FWCI
- 13.11
- Type
- journal article
- Language
- en
- Open access
- hybrid
- doi.org
- Licence
- Locations
- Molecular Therapy (publishedVersion, cc-by-nc-nd)
- PubMed (publishedVersion)
- PubMed Central (acceptedVersion)
- Carolina Digital Repository (University of North Carolina at Chapel Hill) (submittedVersion)
- UNC Libraries
- Funders
- National Institutes of Health
- National Heart, Lung, and Blood Institute (P01HL051818, HL51818)
- National Institute of Allergy and Infectious Diseases (R01AI072176)
- National Institute of Neurological Disorders and Stroke (NS 35633, NS59518, R21NS059518, R01NS035633)
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (R21AR055712, AR055712)
- 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
- Physical Sciences › Materials Science › Biomaterials › Nanoparticle-Based Drug Delivery
- Keywords
- adeno-associated virus, DNA shuffling, directed evolution, heparan sulfate receptor, capsid structure, neutralizing antibodies, gene delivery vectors, biological nanoparticles, AAV serotypes
- MeSH terms
- Dependovirus, Animals, Antibodies, Brain, Capsid, Genetic Vectors, Cricetinae, Humans, Liver, Melanoma, Mice, Inbred BALB C, Primates, Transduction, Genetic, Gene Library, Genome, Viral, Muscle, Skeletal, Directed Molecular Evolution, DNA Shuffling, Mice, Virus Internalization, Nanoparticles
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