The DOI matches its Crossref record; 10 of the 10 references it cites were found in a registry or an index.
Self-Inactivating Lentiviral Vectors with Enhanced Transgene Expression as Potential Gene Transfer System in Parkinson's Disease
Open accessAbstract
Glial cell line-derived neurotrophic factor (GDNF) is able to protect dopaminergic neurons against various insults and constitutes therefore a promising candidate for the treatment of Parkinson's disease. Lentiviral vectors that infect quiescent neuronal cells may allow the localized delivery of GDNF, thus avoiding potential side effects related to the activation of other brain structures. To test this hypothesis in a setting ensuring both maximal biosafety and optimal transgene expression, a self-inactivating (SIN) lentiviral vector was modified by insertion of the posttranscriptional regulatory element of the woodchuck hepatitis virus, and particles were produced with a multiply attenuated packaging system. After a single injection of 2 microl of a lacZ-expressing vector (SIN-W-LacZ) in the substantia nigra of adult rats, an average of 40.1 +/- 6.0% of the tyrosine hydroxylase (TH)-positive neurons were transduced as compared with 5.0 +/- 2.1% with the first-generation lentiviral vector. Moreover, the SIN-W vector expressing GDNF under the control of the mouse phosphoglycerate kinase 1 (PGK) promoter was able to protect nigral dopaminergic neurons after medial forebrain bundle axotomy. Expression of hGDNF in the nanogram range was detected in extracts of mesencephalon of animals injected with an SIN-W-PGK-GDNF vector, whereas it was undetectable in animals injected with a control vector. Lentiviral vectors with enhanced expression and safety features further establish the potential use of these vectors for the local delivery of bioactive molecules into defined structures of the central nervous system.
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Cites
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In Vivo Gene Delivery and Stable Transduction of Nondividing Cells by a Lentiviral Vector
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Self-citationA Third-Generation Lentivirus Vector with a Conditional Packaging System
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Self-citationMultiply attenuated lentiviral vector achieves efficient gene delivery in vivo
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Self-citationDevelopment of a self-inactivating lentivirus vector
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Cited by
Mazzulli, J. R., Xu, Y.-H., Sun, Y., Knight, A. L., McLean, P. J., Caldwell, G. A., Sidransky, E., Grabowski, G. A., & Krainc, D. (2011) · Cell · doi:10.1016/j.cell.2011.06.001 · Cited by 1,341
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Self-citationThe C9orf72 protein interacts with Rab1a and the ULK1 complex to regulate initiation of autophagy
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Isolation of Multipotent Neural Precursors Residing in the Cortex of the Adult Human Brain
Arsenijevic, Y., Villemure, J.-G., Brunet, J.-F., Bloch, J. J., Déglon, N., Kostic, C., Zurn, A., & Aebischer, P. (2001) · Experimental Neurology · doi:10.1006/exnr.2001.7691 · Cited by 310
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Metadata
- Authors
- Deglon, N., Tseng, J. L., Bensadoun, J.-C., Zurn, A. D., Arsenijevic, Y., Almeida, L. P. D., Zufferey, R., Trono, D., & Aebischer, P.
- Venue
- Human Gene Therapy
- Publisher
- SAGE Publications
- Published
- 2000-01
- Volume
- 11
- Issue
- 1
- Pages
- 179-190
- ISSN
- 1043-0342 (ISSN-L)
- 1557-7422
- OpenAlex
- W2150922307
- Cited by
- 292
- References
- 10
- FWCI
- 15.21
- Type
- journal article
- Language
- en
- Open access
- green
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- Deglon, N., Tseng, J. L., Bensadoun, J.-C., Zurn, A. D., Arsenijevic, Y., Almeida, L. P. D., Zufferey, R., Trono, D., & Aebischer, P. (2000). Self-Inactivating Lentiviral Vectors with Enhanced Transgene Expression as Potential Gene Transfer System in Parkinson's Disease. Human Gene Therapy. https://doi.org/10.1089/10430340050016256
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