Gene therapies have entered clinical trials for several neurological disorders, most notably Parkinson disease. A study in a nonhuman primate model of Parkinson disease has reported motor deficit improvements following the use of a lentiviral vector to restore extracellular dopamine levels. A clinical trial is now underway to evaluate the effectiveness of this approach in humans.
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
Jarraya, B. et al. Dopamine gene therapy for Parkinson's Disease in a nonhuman primate without associated dyskinesia. Science Trans. Med. 1, 1–10 (2009).
Kaplitt, M. G. et al. Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial. Lancet 369, 2097–2105 (2007).
Marks, W. J. et al. Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2-neurturin) to patients with idiopathic Parkinson's disease: an open-label, phase I trial. Lancet Neurol. 7, 400–408 (2008).
Christine, C. W. et al. Safety and tolerability of putaminal AADC gene therapy for Parkinson disease. Neurology doi:10.1212/WNL.0b013e3181c29356.
Muramatsu, S. et al. Behavioral recovery in a primate model of Parkinson's disease by triple transduction of striatal cells with adeno-associated viral vectors expressing dopamine-synthesizing enzymes. Hum. Gene Ther. 13, 345–354 (2002).
Bankiewicz, K. S. et al. Focal striatal dopamine may potentiate dyskinesias in parkinsonian monkeys. Exp. Neurol. 197, 363–372 (2006).
Freed, C. R. et al. Transplantation of embryonic dopamine neurons for severe Parkinson's disease. N. Engl. J. Med. 344, 710–719 (2001).
Olanow, C. W. et al. A double-blind controlled trial of bilateral fetal nigral transplantation in Parkinson's disease. Ann. Neurol. 54, 403–414 (2003).
Ma, Y. et al. Dyskinesia after fetal cell transplantation for parkinsonism: a PET study. Ann. Neurol. 52, 628–634 (2002).
Author information
Authors and Affiliations
Ethics declarations
Competing interests
M. G. Kaplitt acts as a consultant for and receives grant support from Neurologix. He also is a stockholder and patent holder with this company.
Rights and permissions
About this article
Cite this article
Kaplitt, M. Another player in gene therapy for Parkinson disease. Nat Rev Neurol 6, 7–8 (2010). https://doi.org/10.1038/nrneurol.2009.214
Issue Date:
DOI: https://doi.org/10.1038/nrneurol.2009.214
This article is cited by
-
A novel detector using a fluorescent sensor array and discrimination of pesticides
Research on Chemical Intermediates (2016)
-
Lentiviral Vector Mediates Exogenous Gene Expression in Adult Rat DRG Following Peripheral Nerve Remote Delivery
Journal of Molecular Neuroscience (2012)
-
Delayed Dominant-Negative TNF Gene Therapy Halts Progressive Loss of Nigral Dopaminergic Neurons in a Rat Model of Parkinson's Disease
Molecular Therapy (2011)
-
Translational benefits of gene therapy to date
Clinical Oncology and Cancer Research (2011)
