The DOI matches its Crossref record; 33 of the 33 references it cites were found in a registry or an index.
Viral vectors for gene delivery to the central nervous system
Open accessAbstract
The potential benefits of gene therapy for neurological diseases such as Parkinson’s, Amyotrophic Lateral Sclerosis (ALS), Epilepsy, and Alzheimer’s are enormous. Even a delay in the onset of severe symptoms would be invaluable to patients suffering from these and other diseases. Significant effort has been placed in developing vectors capable of delivering therapeutic genes to the CNS in order to treat neurological disorders. At the forefront of potential vectors, viral systems have evolved to efficiently deliver their genetic material to a cell. The biology of different viruses offers unique solutions to the challenges of gene therapy, such as cell targeting, transgene expression and vector production. It is important to consider the natural biology of a vector when deciding whether it will be the most effective for a specific therapeutic function. In this review, we outline desired features of the ideal vector for gene delivery to the CNS and discuss how well available viral vectors compare to this model. Adeno-associated virus, retrovirus, adenovirus and herpesvirus vectors are covered. Focus is placed on features of the natural biology that have made these viruses effective tools for gene delivery with emphasis on their application in the CNS. Our goal is to provide insight into features of the optimal vector and which viral vectors can provide these features.
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Cites
In Vivo Gene Delivery and Stable Transduction of Nondividing Cells by a Lentiviral Vector
Naldini, L., Blömer, U., Gallay, P., Ory, D., Mulligan, R., Gage, F. H., Verma, I. M., & Trono, D. (1996) · Science · doi:10.1126/science.272.5259.263 · Cited by 4,903
A Third-Generation Lentivirus Vector with a Conditional Packaging System
Dull, T., Zufferey, R., Kelly, M., Mandel, R. J., Nguyen, M., Trono, D., & Naldini, L. (1998) · Journal of Virology · doi:10.1128/jvi.72.11.8463-8471.1998 · Cited by 3,341
Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo
Zufferey, R., Nagy, D., Mandel, R. J., Naldini, L., & Trono, D. (1997) · Nature Biotechnology · doi:10.1038/nbt0997-871 · Cited by 1,977
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
Development of a self-inactivating lentivirus vector
Miyoshi, H., Blömer, U., Takahashi, M., Gage, F. H., & Verma, I. M. (1998) · Journal of Virology · doi:10.1128/jvi.72.10.8150-8157.1998 · Cited by 1,196
A phase 1 clinical trial of nerve growth factor gene therapy for Alzheimer disease
Tuszynski, M. H., Thal, L., Pay, M., Salmon, D. P., U, H. S., Bakay, R., Patel, P., Blesch, A., Vahlsing, H. L., Ho, G., et al. (2005) · Nature Medicine · doi:10.1038/nm1239 · Cited by 1,050
Effect of genome size on AAV vector packaging
Wu, Z., Yang, H., & Colosi, P. (2010) · Molecular Therapy · doi:10.1038/mt.2009.255 · Cited by 1,029
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-citationMcCarty, D., Monahan, P., & Samulski, R. (2001) · Gene Therapy · doi:10.1038/sj.gt.3301514 · Cited by 735
Self-citationHIV-1 Gag proteins: diverse functions in the virus life cycle
Freed, E. O. (1998) · Virology · doi:10.1006/viro.1998.9398 · Cited by 717
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Lee, C. S., Bishop, E. S., Zhang, R., Yu, X., Farina, E. M., Yan, S., Zhao, C., Zeng, Z., Shu, Y., Wu, X., et al. (2017) · Genes & Diseases · doi:10.1016/j.gendis.2017.04.001 · Cited by 691
(2013) · PubMed · Cited by 308
Gene therapy for the CNS using AAVs: The impact of systemic delivery by AAV9
Saraiva, J., Nobre, R. J., & Pereira de Almeida, L. (2016) · Journal of Controlled Release · doi:10.1016/j.jconrel.2016.09.011 · Cited by 201
Lipid nanoparticle-mediated drug delivery to the brain
Khare, P., Edgecomb, S. X., Hamadani, C. M., Tanner, E. E. L., & S Manickam, D. (2023) · Advanced Drug Delivery Reviews · doi:10.1016/j.addr.2023.114861 · Cited by 187
The advent of AAV9 expands applications for brain and spinal cord gene delivery
Dayton, R. D., Wang, D. B., & Klein, R. L. (2012) · Expert Opinion on Biological Therapy · doi:10.1517/14712598.2012.681463 · Cited by 144
Methods for Gene Transfer to the Central Nervous System
Kantor, B., Bailey, R. M., Wimberly, K., Kalburgi, S. N., & Gray, S. J. (2014) · Advances in Genetics · doi:10.1016/b978-0-12-800149-3.00003-2 · Cited by 109
Self-citationBailey, R. M., Rozenberg, A., & Gray, S. J. (2020) · Brain Research · doi:10.1016/j.brainres.2020.146832 · Cited by 83
Self-citationHighly PEGylated DNA Nanoparticles Provide Uniform and Widespread Gene Transfer in the Brain
Mastorakos, P., Zhang, C., Berry, S., Oh, Y., Lee, S., Eberhart, C. G., Woodworth, G. F., Suk, J. S., & Hanes, J. (2015) · Advanced Healthcare Materials · doi:10.1002/adhm.201400800 · Cited by 80
rAAV-compatible MiniPromoters for restricted expression in the brain and eye
de Leeuw, C. N., Korecki, A. J., Berry, G. E., Hickmott, J. W., Lam, S. L., Lengyell, T. C., Bonaguro, R. J., Borretta, L. J., Chopra, V., Chou, A. Y., et al. (2016) · Molecular Brain · doi:10.1186/s13041-016-0232-4 · Cited by 76
Fu, C., Xiang, Y., Li, X., & Fu, A. (2018) · Materials Science and Engineering: C · doi:10.1016/j.msec.2017.12.029 · Cited by 48
On the development of optical peripheral nerve interfaces
Anderson, H., & Weir, R. F. (2019) · Neural Regeneration Research · doi:10.4103/1673-5374.245461 · Cited by 14
Serum/glucocorticoid regulated kinase 1 (SGK1) in neurological disorders: pain or gain
Howard, P. G., Zou, P., Zhang, Y., Huang, F., Tesic, V., Wu, C. Y.-C., & Lee, R. H.-C. (2024) · Experimental Neurology · doi:10.1016/j.expneurol.2024.114973 · Cited by 13
Epstein-Barr virus-derived vector suitable for long-term expression in neurons
Kiyosue, K., & Miwa, Y. (2020) · Heliyon · doi:10.1016/j.heliyon.2020.e03504 · Cited by 8
Metadata
- Authors
- Lentz, T. B., Gray, S. J., & Samulski, R. J.
- Venue
- Neurobiology of Disease
- Publisher
- Elsevier BV
- Published
- 2012-11
- Volume
- 48
- Issue
- 2
- Pages
- 179-188
- ISSN
- 0969-9961 (ISSN-L)
- OpenAlex
- W2068666140
- Cited by
- 271
- References
- 33
- FWCI
- 7.86
- Type
- journal article
- Language
- en
- Open access
- green
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