The DOI matches its Crossref record; 12 of the 12 references it cites were found in a registry or an index.
Recombinant AAV-mediated gene delivery to the central nervous system
Abstract
Various regions of the brain have been successfully transduced by recombinant adeno-associated virus (rAAV) vectors with no detected toxicity. When using the cytomegalovirus immediate early (CMV) promoter, a gradual decline in the number of transduced cells has been described. In contrast, the use of cellular promoters such as the neuron-specific enolase promoter or hybrid promoters such as the chicken beta-actin/CMV promoter resulted in sustained transgene expression. The cellular tropism of rAAV-mediated gene transfer in the central nervous system (CNS) varies depending on the serotype used. Serotype 2 vectors preferentially transduce neurons whereas rAAV5 and rAAV1 transduce both neurons and glial cells. Recombinant AAV4-mediated gene transfer was inefficient in neurons and glial cells of the striatum (the only structure tested so far) but efficient in ependymal cells. No inflammatory response has been described following rAAV2 administration to the brain. In contrast, antibodies to AAV2 capsid and transgene product were elicited but no reduction of transgene expression was observed and readministration of vector without loss of efficiency was possible from 3 months after the first injection. Based on the success of pioneer work performed with marker genes, various strategies for therapeutic gene delivery were designed. These include enzyme replacement in lysosomal storage diseases, Canavan disease and Parkinson's disease; delivery of neuroprotective factors in Parkinson's disease, Huntington disease, Alzheimer's disease, amyotrophic lateral sclerosis, ischemia and spinal cord injury; as well as modulation of neurotransmission in epilepsy and Parkinson's disease. Several of these strategies have demonstrated promising results in relevant animal models. However, their implementation in the clinics will probably require a tight regulation and a specific targeting of therapeutic gene expression which still demands further developments of the vectors.
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Cites
Direct brain infusion of glial cell line–derived neurotrophic factor in Parkinson disease
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In vivo model of adeno-associated virus vector persistence and rescue
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Cited by
Potential therapeutic uses of BDNF in neurological and psychiatric disorders
Nagahara, A. H., & Tuszynski, M. H. (2011) · Nature Reviews Drug Discovery · doi:10.1038/nrd3366 · Cited by 867
Burger, C., Gorbatyuk, O. S., Velardo, M. J., Peden, C. S., Williams, P., Zolotukhin, S., Reier, P. J., Mandel, R. J., & Muzyczka, N. (2004) · Molecular Therapy · doi:10.1016/j.ymthe.2004.05.024 · Cited by 741
Target-based drug discovery: is something wrong?
Sams-Dodd, F. (2005) · Drug Discovery Today · doi:10.1016/s1359-6446(04)03316-1 · Cited by 522
Gray, S. J., Foti, S. B., Schwartz, J. W., Bachaboina, L., Taylor-Blake, B., Coleman, J., Ehlers, M. D., Zylka, M. J., McCown, T. J., & Samulski, R. J. (2011) · Human Gene Therapy · doi:10.1089/hum.2010.245 · Cited by 420
Ependymal cells: biology and pathology
Del Bigio, M. R. (2010) · Acta Neuropathologica · doi:10.1007/s00401-009-0624-y · Cited by 386
Metadata
- Authors
- Tenenbaum, L., Chtarto, A., Lehtonen, E., Velu, T., Brotchi, J., & Levivier, M.
- Venue
- The Journal of Gene Medicine
- Publisher
- Wiley
- Published
- 2004-02
- Volume
- 6
- Issue
- S1
- Pages
- S212-S222
- ISSN
- 1099-498X (ISSN-L)
- 1521-2254
- OpenAlex
- W2114618955
- Cited by
- 204
- References
- 12
- FWCI
- 8.42
- Type
- journal article
- Language
- en
- Open access
- closed
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