Viral Vectors for Gene Transfer
Abstract
Viral vectors are a promising tool for effective delivery of genetic material into cells. They take advantage of the natural ability of a virus to deliver a genetic payload into cells while being genetically modified such that their ability to replicate is crippled or removed. Here, an updated overview of routinely used viral vectors, including adeno‐associated viruses (AAV), retroviruses/lentiviruses, and adenoviruses (Ads), is provided, as well as perspectives on their advantages and disadvantages in research and gene therapy. © 2018 by John Wiley & Sons, Inc.
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AAV is one of the most common vectors used in gene therapy.
“AAV is one of the most common vectors used in gene therapy.”
From Viral Vector Systems for Gene Therapy: A Comprehensive Literature Review of Progress and Biosafety Challenges (2020) · cites this paper as [19]
AAV vectors have a small packaging capacity that is generally considered to be less than 5 kb.
“Another substantial limitation of AAV vectors is their small packaging capacity that is generally considered to be <5 kb.”
From Viral Vector Systems for Gene Therapy: A Comprehensive Literature Review of Progress and Biosafety Challenges (2020) · cites this paper as [19]
The AV vector now represents 1 of the 3 major viral vector categories in the gene therapy toolbox, along with AAV and lenti/retrovirus vector systems.
“The AV vector now represents 1 of the 3 major viral vector categories in the gene therapy toolbox, along with AAV and lenti/retrovirus vector systems.”
From Viral Vector Systems for Gene Therapy: A Comprehensive Literature Review of Progress and Biosafety Challenges (2020) · cites this paper as [19]
BSL-2 is appropriate for many constructs of HSV.
“BSL-2 is appropriate for many constructs of HSV.”
From Viral Vector Systems for Gene Therapy: A Comprehensive Literature Review of Progress and Biosafety Challenges (2020) · cites this paper as [19]
Cites
Gene therapy returns to centre stage
Naldini, L. (2015) · Nature · doi:10.1038/nature15818 · Cited by 1,184 · see citing works
Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant
George, L. A., Sullivan, S. K., Giermasz, A., Rasko, J. E. J., Samelson-Jones, B. J., Ducore, J., Cuker, A., Sullivan, L. M., Majumdar, S., Teitel, J., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1708538 · Cited by 762 · see citing works
Self-citationAAV-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 582 · see citing works
Brown, B. D., Gentner, B., Cantore, A., Colleoni, S., Amendola, M., Zingale, A., Baccarini, A., Lazzari, G., Galli, C., & Naldini, L. (2007) · Nature Biotechnology · doi:10.1038/nbt1372 · Cited by 559 · see citing works
Zhong, L., Li, B., Mah, C. S., Govindasamy, L., Agbandje-McKenna, M., Cooper, M., Herzog, R. W., Zolotukhin, I., Warrington, K. H., Weigel-Van Aken, K. A., et al. (2008) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.0802866105 · Cited by 558 · see citing works
An essential receptor for adeno-associated virus infection
Pillay, S., Meyer, N. L., Puschnik, A. S., Davulcu, O., Diep, J., Ishikawa, Y., Jae, L. T., Wosen, J. E., Nagamine, C. M., Chapman, M. S., et al. (2016) · Nature · doi:10.1038/nature16465 · Cited by 532 · see citing works
CAR T-cell Therapy: A New Era in Cancer Immunotherapy
Miliotou, Α. N., & Papadopoulou, L. C. (2018) · Current Pharmaceutical Biotechnology · doi:10.2174/1389201019666180418095526 · Cited by 479 · see citing works
The AAV Vector Toolkit: Poised at the Clinical Crossroads
Asokan, A., Schaffer, D. V., & Samulski, R. J. (2012) · Molecular Therapy · doi:10.1038/mt.2011.287 · Cited by 463 · see citing works
Bennett, J., Wellman, J., Marshall, K. A., McCague, S., Ashtari, M., DiStefano-Pappas, J., Elci, O. U., Chung, D. C., Sun, J., Wright, J. F., et al. (2016) · The Lancet · doi:10.1016/s0140-6736(16)30371-3 · Cited by 461 · see citing works
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 · see citing works
Multiplex CRISPR/Cas9-based genome engineering from a single lentiviral vector
Kabadi, A. M., Ousterout, D. G., Hilton, I. B., & Gersbach, C. A. (2014) · Nucleic Acids Research · doi:10.1093/nar/gku749 · Cited by 371 · see citing works
Brunetti‐Pierri, N., Palmer, D. J., Beaudet, A. L., et al. (2004) · Human Gene Therapy · doi:10.1089/10430340460732445 · Cited by 257 · see citing works
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 · see citing works
Strunze, S., Engelke, M., Wang, I.-H., Puntener, D., Boucke, K., Schleich, S., Way, M., Schoenenberger, P., Burckhardt, C. J., & Greber, U. (2011) · Cell Host and Microbe · doi:10.1016/j.chom.2011.08.010 · Cited by 209 · see citing works
Rivella, S., Callegari, J., May, C., Tan, C. W., & Sadelain, M. (2000) · Journal of Virology · doi:10.1128/jvi.74.10.4679-4687.2000 · Cited by 201 · see citing works
Molecular signatures of disease brain endothelia provide new sites for CNS-directed enzyme therapy
Chen, Y. H., Chang, M., & Davidson, B. L. (2009) · Nature Medicine · doi:10.1038/nm.2025 · Cited by 168 · see citing works
Self-citationAdenovirus receptors: implications for tropism, treatment and targeting
Arnberg, N. (2009) · Reviews in Medical Virology · doi:10.1002/rmv.612 · Cited by 145 · see citing works
Katz, M. L., Tecedor, L., Chen, Y., Williamson, B. G., Lysenko, E., Wininger, F. A., Young, W. M., Johnson, G. C., Whiting, R. E. H., Coates, J. R., et al. (2015) · Science Translational Medicine · doi:10.1126/scitranslmed.aac6191 · Cited by 134 · see citing works
Self-citationEarley, L. F., Powers, J. M., Adachi, K., Baumgart, J. T., Meyer, N. L., Xie, Q., Chapman, M. S., & Nakai, H. (2017) · Journal of Virology · doi:10.1128/jvi.01980-16 · Cited by 108 · see citing works
Leukemia induction after a single retroviral vector insertion in Evi1 or Prdm16
Modlich, U., Schambach, A., Brugman, M. H., Wicke, D. C., Knoess, S., Li, Z., Maetzig, T., Rudolph, C., Schlegelberger, B., & Baum, C. (2008) · Leukemia · doi:10.1038/leu.2008.118 · Cited by 98 · see citing works
The Inside Out of Lentiviral Vectors
Durand, S., & Cimarelli, A. (2011) · Viruses · doi:10.3390/v3020132 · Cited by 97 · see citing works
Host determinants of adeno-associated viral vector entry
Pillay, S., & Carette, J. E. (2017) · Current Opinion in Virology · doi:10.1016/j.coviro.2017.06.003 · Cited by 94 · see citing works
Pseudotyped Lentiviral Vectors: One Vector, Many Guises
Joglekar, A. V., & Sandoval, S. (2017) · Human Gene Therapy Methods · doi:10.1089/hgtb.2017.084 · Cited by 88 · see citing works
Recent advances in oncolytic adenovirus therapies for cancer
Shaw, A. R., & Suzuki, M. (2016) · Current Opinion in Virology · doi:10.1016/j.coviro.2016.06.009 · Cited by 87 · see citing works
Delivering Transgenic DNA Exceeding the Carrying Capacity of AAV Vectors
Hirsch, M. L., Wolf, S., & Samulski, R. J. (2015) · Methods in molecular biology · doi:10.1007/978-1-4939-3271-9_2 · Cited by 84 · see citing works
Retroviruses: Molecular Biology, Genomics and Pathogenesis
Telesnitsky, A. (2010) · Future Virology · doi:10.2217/fvl.10.43 · Cited by 81 · see citing works
Interactions between Retroviruses and the Host Cell Genome
Poletti, V., & Mavilio, F. (2017) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2017.10.001 · Cited by 81 · see citing works
MECP2 Isoform-Specific Vectors with Regulated Expression for Rett Syndrome Gene Therapy
Rastegar, M., Hotta, A., Pasceri, P., Makarem, M., Cheung, A. Y. L., Elliott, S., Park, K. J., Adachi, M., Jones, F. S., Clarke, I. D., et al. (2009) · PLoS ONE · doi:10.1371/journal.pone.0006810 · Cited by 79 · see citing works
RNAi prevents and reverses phenotypes induced by mutant human ataxin‐1
Keiser, M. S., Monteys, A. M., Corbau, R., Gonzalez‐Alegre, P., & Davidson, B. L. (2016) · Annals of Neurology · doi:10.1002/ana.24789 · Cited by 57 · see citing works
Self-citationDoering, C. B., Denning, G., Shields, J. E., Fine, E. J., Parker, E. T., Srivastava, A., Lollar, P., & Spencer, H. T. (2018) · Human Gene Therapy · doi:10.1089/hum.2018.137 · Cited by 56 · see citing works
Regulating nociceptive transmission by VG luT2‐expressing spinal dorsal horn neurons
Wang, L., Chen, S., Ma, H., Chen, H., Hittelman, W. N., & Pan, H. (2018) · Journal of Neurochemistry · doi:10.1111/jnc.14588 · Cited by 48 · see citing works
Scherer, J., & Vallee, R. B. (2011) · Viruses · doi:10.3390/v3081417 · Cited by 46 · see citing works
Future Prospects for the Development of Cost-Effective Adenovirus Vaccines
Fougeroux, C. E., & Holst, P. J. (2017) · International Journal of Molecular Sciences · doi:10.3390/ijms18040686 · Cited by 45 · see citing works
The retroviral vector family: something for everyone
Elsner, C., & Bohne, J. (2017) · Virus Genes · doi:10.1007/s11262-017-1489-0 · Cited by 36 · cited 2× · see citing works
“Furthermore, tat is eliminated through the addition of a chimeric 5 LTR fused to a heterologous promoter on the vector plasmid (Elsner & Bohne, 2017).”
Gene therapy with helper-dependent adenoviral vectors: lessons from studies in large animal models
Brunetti‐Pierri, N., & Ng, P. (2017) · Virus Genes · doi:10.1007/s11262-017-1471-x · Cited by 33 · see citing works
Ungari, S., Montepeloso, A., Morena, F., et al. (2015) · Molecular Therapy — Methods & Clinical Development · doi:10.1038/mtm.2015.38 · Cited by 32 · see citing works
High Capsid–Genome Correlation Facilitates Creation of AAV Libraries for Directed Evolution
Nonnenmacher, M. E., van Bakel, H., Hajjar, R. J., & Weber, T. (2015) · Molecular Therapy · doi:10.1038/mt.2015.3 · Cited by 32 · see citing works
Directed Evolution of Adeno-Associated Virus (AAV) as Vector for Muscle Gene Therapy
Yang, L., Li, J., & Xiao, X. (2010) · Methods in molecular biology · doi:10.1007/978-1-61737-982-6_8 · Cited by 31 · see citing works
Pseudotyped Lentiviral Vectors for Retrograde Gene Delivery into Target Brain Regions
Kobayashi, K., Inoue, K., Tanabe, S., Kato, S., Takada, M., & Kobayashi, K. (2017) · Frontiers in Neuroanatomy · doi:10.3389/fnana.2017.00065 · Cited by 30 · see citing works
Leggiero, E., Astone, D., Cerullo, V., Lombardo, B., Mazzaccara, C., Labruna, G., Sacchetti, L., Salvatore, F., Croyle, M. A., & Pastore, L. (2013) · Gene Therapy · doi:10.1038/gt.2013.38 · Cited by 28 · see citing works
Viral vector-based tools advance knowledge of basal ganglia anatomy and physiology
Sizemore, R. J., Seeger-Armbruster, S., Hughes, S. M., & Parr‐Brownlie, L. C. (2016) · Journal of Neurophysiology · doi:10.1152/jn.01131.2015 · Cited by 23 · see citing works
Novel Vector Construction Based on Alternative Adenovirus Types via Homologous Recombination
Zhang, W., Fu, J., & Ehrhardt, A. (2018) · Human Gene Therapy Methods · doi:10.1089/hgtb.2018.044 · Cited by 21 · see citing works
Unzu, C., Melero, I., Hervás‐Stubbs, S., Sampedro, A., Mancheño, U., Morales‐Kastresana, A., Serrano-Mendioroz, I., de Salamanca, R. E., Benito, A., & Fontanellas, A. (2015) · Gene Therapy · doi:10.1038/gt.2015.64 · Cited by 16 · see citing works
Adenovirus Composition, Structure, and Biophysical Properties
(2016) · WORLD SCIENTIFIC eBooks · doi:10.1142/9789813109803_0002 · Cited by 3 · see citing works
Hirsch, M. L. (2019) · Carolina Digital Repository (University of North Carolina at Chapel Hill) · doi:10.17615/5baf-f868 · Cited by 1 · see citing works
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Gebre, M. S., Brito, L. A., Tostanoski, L. H., Edwards, D. K., Carfi, A., & Barouch, D. H. (2021) · Cell · doi:10.1016/j.cell.2021.02.030 · Cited by 347 · see citing works
Ghosh, S., Brown, A. M., Jenkins, C., & Campbell, K. (2020) · Applied Biosafety · doi:10.1177/1535676019899502 · Cited by 218 · see citing works
Sun, W., Xing, C., Zhao, L., Zhao, P., Yang, G., & Yuan, L. (2020) · Molecular Therapy Nucleic Acids · doi:10.1016/j.omtn.2020.03.016 · Cited by 62 · see citing works
- Authors
- Chen, Y. H., Keiser, M. S., & Davidson, B. L.
- Affiliations
- Children's Hospital of Philadelphia (US)
- Corresponding author
- Davidson, Beverly L.
- Venue
- Current Protocols in Mouse Biology
- Publisher
- Wiley
- Published
- 2018-11-28
- Volume
- 8
- Issue
- 4
- Pages
- e58-e58
- ISSN
- 2161-2617 (ISSN-L)
- OpenAlex
- W2903362002
- Cited by
- 146
- References
- 45
- Citations by year
- 2026: 12 · 2025: 15 · 2024: 26 · 2023: 20 · 2022: 31 · 2021: 23 · 2020: 17 · 2019: 2
- FWCI
- 1.47
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Current Protocols in Mouse Biology (publishedVersion)
- PubMed (publishedVersion)
- 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
- viral vectors, gene transfer, gene therapy, adeno-associated virus, retroviruses, lentivirus, adenovirus, viral vector design, gene delivery
- MeSH terms
- Dependovirus, Animals, Disease Models, Animal, Genetic Vectors, Genetic Therapy, Mice
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