Current therapies for Parkinson disease focus mainly on symptomatic relief, but gene therapy approaches may offer the prospect of halting or reversing the progress of the disease process itself. A recent study provides evidence that sustained delivery of glial cell line-derived neurotrophic factor to the nigrostriatal system provides neuroprotection and functional recovery in a primate model of Parkinson disease.
This is a preview of subscription content, access via your institution
Access options
Subscribe to this journal
Receive 12 print issues and online access
$209.00 per year
only $17.42 per issue
Buy this article
- Purchase on Springer Link
- Instant access to full article PDF
Prices may be subject to local taxes which are calculated during checkout
References
Kordower, J.H. et al. Neurodegeneration prevented by lentiviral vector delivery of GDNF in primate models of Parkinson's disease. Science (in the press).
Lin, L.-F.H., Doherty, D.H., Lile, J.D., Bektesh, S. & Collins, F. GDNF: a glial cell line-derived neurotrophic factor for midbrain dopaminergic neurons. Science 260, 1130–1132 (1993).
Björklund, A., Rosenblad, C., Winkler, C. & Kirik, D. Studies on neuroprotective and regenerative effects of GDNF in a partial lesion model of Parkinson's disease. Neurobiol. Dis. 4, 186–200 (1997).
Gash, D.M., Zhang, Z.M. & Gerhardt, G. Neuroprotective and neurorestorative properties of GDNF. Ann. Neurol. 44, S121–S125 (1998).
Kordower, J.H. et al. Clinicopathological findings following intraventricular glial-derived neurotrophic factor treatment in a patient with Parkinson's disease. Ann. Neurol. 46, 419–424 (1999).
Choi-Lundberg, D.L. et al. Dopaminergic neurons protected from degeneration by GDNF gene therapy. Science 275, 838–841 (1997).
Kirik, D., Rosenblad, C., Björklund, A. & Mandel, R.J. Long-term rAAV-mediated gene transfer of GDNF in the rat Parkinson's model: intrastriatal but not intranigral transduction promotes functional regeneration in the lesioned nigrostriatal system. J. Neurosci. 20, 4686–4700 (2000).
Deglon, N. et al. Self-inactivating lentiviral vectors with enhanced transgene expression as potential gene transfer system in Parkinson's disease. Hum. Gene Ther. 11, 179–190 (2000).
Rosenblad, C. et al. In vivo protection of nigral dopamine neurons by lentiviral gene transfer of the novel GDNF-family member Neublastin/Artemin. Mol. Cell Neurosci. 15, 199–214 (2000).
Blomer, U.L. et al. Highly efficient and sustained gene transfer in adult neurons with a lentivirus vector. J. Virol. 71, 6641–6649 (1997).
Trono, D. Lentiviral vectors: turning a deadly foe into a therapeutic agent. Gene Ther. 7, 20–23 (2000).
Morrish, P.K., Rakshi, J.S., Bailey, D.L., Sawle, G.V. & Brooks, D.J. Measuring the rate of progression and estimating the preclinical period of Parkinson's disease with 18F-dopa PET. J. Neurol. Neurosurg. Psychiatry 64, 314–319 (1998).
Author information
Authors and Affiliations
Rights and permissions
About this article
Cite this article
Björklund, A., Lindvall, O. Parkinson disease gene therapy moves toward the clinic. Nat Med 6, 1207–1208 (2000). https://doi.org/10.1038/81291
Issue Date:
DOI: https://doi.org/10.1038/81291
This article is cited by
-
Comparative studies on cellular gene regulation by HIV-1 based vectors: implications for quality control of vector production
Gene Therapy (2005)
-
Gene therapy in Parkinson?s disease
Cell and Tissue Research (2004)
-
Neuroprotective and neurorestorative strategies for Parkinson's disease
Nature Neuroscience (2002)
