The DOI matches its Crossref record; 186 of the 186 references it cites were found in a registry or an index.
Basic Biology of Adeno-Associated Virus (AAV) Vectors Used in Gene Therapy
Abstract
Adeno-associated virus (AAV) based vectors have emerged as important tools for gene therapy in humans. The recent successes seen in Phase I/II clinical trials have also highlighted the issues related to the host and vector-related immune response that preclude the universal application of this promising vector system. A fundamental insight into the biological mechanisms by which AAV infects the host cell and a thorough understanding of the immediate and long-lived cellular responses to AAV infection is likely to offer clues and help design better intervention strategies to improve the therapeutic efficiency of AAV vectors. This article reviews the biology of AAV-host cellular interactions and outlines their application in the development of novel and improved AAV vector systems.
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“Thus, AAV viral vectors provide valuable opportunities for clinical applications in the treatment of neurodegenerative diseases [5, 6].”
From Designing and optimizing AAV-mediated gene therapy for neurodegenerative diseases: from bench to bedside (2024) · cites this paper as [6]
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Cites
Manno, C. S., Pierce, G. F., Arruda, V. R., Glader, B., Ragni, M., Rasko, J. J. E., Ozelo, M. C., Hoots, K., Blatt, P., Konkle, B., et al. (2006) · Nature Medicine · doi:10.1038/nm1358 · Cited by 2,163
Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis
Maguire, A. M., Simonelli, F., Pierce, E. A., Pugh, E. N., Mingozzi, F., Bennicelli, J., Banfi, S., Marshall, K. A., Testa, F., Surace, E. M., et al. (2008) · New England Journal of Medicine · doi:10.1056/nejmoa0802315 · Cited by 2,145
Effect of gene therapy on visual function in Leber’s congenital amaurosis
Bainbridge, J. W. B., Smith, A. J., Barker, S. S., Robbie, S., Henderson, R., Balaggan, K., Viswanathan, A., Holder, G. E., Stockman, A., Tyler, N., et al. (2008) · New England Journal of Medicine · doi:10.1056/nejmoa0802268 · Cited by 1,924
Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B
Nathwani, A. C., Tuddenham, E. G. D., Rangarajan, S., Rosales, C., McIntosh, J., Linch, D. C., Chowdary, P., Riddell, A., Pie, A. J., Harrington, C., et al. (2011) · New England Journal of Medicine · doi:10.1056/nejmoa1108046 · Cited by 1,864
Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy
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Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection
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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 · cited 1×
“Based on in vivo studies, vectors specific to retina (AAV2, AAV4 and AAV5) [64-68], brain (AAV2, AAV5, AAV8, AAV9 and AAVrh10) [69-78], cardiac tissue (AAV1, AAV2, AAV6, AAV8 and AAV9) [79-83], liver (AAV1, AAV2, AAV5, AAV6, AAV7, AAV8, AAV9 and AAV10) [49, 84-93] and lungs (AAV1, AAV2, AAV5 and AAV9) [86-89, 92-98] h…”
Summerford, C., & Samulski, R. J. (1998) · Journal of Virology · doi:10.1128/jvi.72.2.1438-1445.1998 · Cited by 1,409
Gene therapy restores vision in a canine model of childhood blindness
Acland, G. M., Aguirre, G. D., Ray, J., Zhang, Q., Aleman, T. S., Cideciyan, A. V., Pearce-Kelling, S. E., Anand, V., Zeng, Y., Maguire, A. M., et al. (2001) · Nature Genetics · doi:10.1038/ng0501-92 · Cited by 1,202 · cited 2×
“Based on in vivo studies, vectors specific to retina (AAV2, AAV4 and AAV5) [64-68], brain (AAV2, AAV5, AAV8, AAV9 and AAVrh10) [69-78], cardiac tissue (AAV1, AAV2, AAV6, AAV8 and AAV9) [79-83], liver (AAV1, AAV2, AAV5, AAV6, AAV7, AAV8, AAV9 and AAV10) [49, 84-93] and lungs (AAV1, AAV2, AAV5 and AAV9) [86-89, 92-98] h…”
Clades of Adeno-Associated Viruses Are Widely Disseminated in Human Tissues
Gao, G., Vandenberghe, L. H., Alvira, M. R., Lu, Y., Calcedo, R., Zhou, X., & Wilson, J. M. (2004) · Journal of Virology · doi:10.1128/jvi.78.12.6381-6388.2004 · Cited by 1,070
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Gene Therapy for Hypophosphatasia in Alpl−/− mice (infantile HPP model) -Toward Clinical Application
Matsumoto, T. (2023) · Nihon Ika Daigaku Igakkai Zasshi · doi:10.1272/manms.19.229
Zou, L. (2024) · Journal of Clinical and Laboratory Research · doi:10.31579/2768-0487/135
Preclinical development of viral vector gene therapies
Newman, M. S., Hudry, E., Brassard, J. A., & Cunningham, J. J. (2024) · Elsevier eBooks · doi:10.1016/b978-0-443-13344-2.00003-1
AAV2 serotype demonstrates the highest transduction efficiency in porcine lung-derived cells
Ju, W. S., Kim, S., Choi, A., Lee, J.-Y., Lee, H., No, J.-G., Lee, S., Oh, K. B., & Yoo, J. G. (2015) · Journal of Animal Reproduciton and Biotechnology · doi:10.12750/jarb.39.4.254 · Cited by 1
Crispr Technology as A Treatment Option for Inherited Neuropathies
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Kaelber, J. T., Barnakov, V., Shen, J., Hernandez, K., Tarbox, H. J., Khan, A., & Escalante, C. R. (2025) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.64898/2025.12.09.693273 · Cited by 1
Exploration of genes and identification of evolutionary evidence in adeno-associated viruses
Lee, C., Min, J., Lim, S., Park, A., Kwak, S., Hwang, S., Park, S., Jang, Y., Cha, S.-Y., Cho, S.-G., et al. (2026) · The Journal of Microbiology · doi:10.71150/jm.2511016
Manz, J., Ruppert, R., Haindl, M., Hubbuch, J. J., & Pschirer, J. (2026) · Molecular Therapy Advances · doi:10.1016/j.omta.2026.201728
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Vandamme, C. (2016) · theses.fr (ABES) · doi:10.70675/371e4bbfze69az417bz960azf9db02287546
Characterization of rAAV vectors packaging in baculovirusinfected insect cells
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Leite, G. D. F. (2017) · theses.fr (ABES) · doi:10.70675/ea0be1b4zfae0z4e90z811cz71c3a7621eb2
Thérapie génique pour l’amyotrophie spinale à l’aide de vecteur AAV
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Modification chimique d’AAV pour la thérapie génique
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Regenerative Strategies for Myocardial Infarction and Their Present Clinical Application
Cruz, J. M. C., Nevado, J. B., Lozano-Gerona, J., Manua, A. J., Agsaoay, M. L. J. S., Pedro, J. V. B. S., Ledesma, D. J. T., Manangan, A. B. G., & Mazahery, A. R. (2026) · Rejuvenation Research · doi:10.1177/15491684261496118
Metadata
- Authors
- Balakrishnan, B., & Jayandharan, G.
- Affiliations
- Bentham Science Publishers (United Arab Emirates) (AE)
- Christian Medical College, Vellore (IN)
- Venue
- Current Gene Therapy
- Publisher
- Bentham Science Publishers Ltd.
- Published
- 2014-05-31
- Volume
- 14
- Issue
- 2
- Pages
- 86-100
- ISSN
- 1566-5232 (ISSN-L)
- OpenAlex
- W2142695353
- Cited by
- 225
- References
- 186
- Citations by year
- 2026: 6 · 2025: 29 · 2024: 22 · 2023: 24 · 2022: 24 · 2021: 28 · 2020: 37 · 2019: 18 · 2018: 13 · 2017: 13 · 2016: 7 · 2015: 1 · 2014: 3
- FWCI
- 5.24
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Current Gene Therapy (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 › Immunology and Microbiology › Immunology › Immunotherapy and Immune Responses
- Keywords
- adeno-associated virus, adeno-associated virus vectors, gene therapy vectors, host immune response, vector immunogenicity, vector design
- MeSH terms
- Dependovirus, Genetic Vectors, Humans, Genetic Therapy, Clinical Trials, Phase I as Topic, Clinical Trials, Phase II as Topic, Gene Transfer Techniques, Host-Pathogen Interactions
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