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Parkinson's disease: gene therapies
Philippe G Coune1, Bernard L Schneider, Patrick Aebischer
1Neurodegenerative Studies Laboratory, Brain Mind Institute, Ecole Polytechnique Fédérale de Lausanne (EPFL), CH-1015 Lausanne, Switzerland.
Cold Spring Harbor Perspectives in Medicine
|April 5, 2012
Summary
Gene therapy using viral vectors shows promise for Parkinson's disease by targeting neurons. Future strategies aim to modify the disease, not just symptoms, offering hope for slowing progression.
Area of Science:
- Neuroscience
- Genetics
- Medical Technology
Background:
- Parkinson's disease (PD) is a neurodegenerative disorder primarily affecting motor function due to nigrostriatal degeneration.
- Current treatments focus on alleviating motor symptoms but do not halt disease progression.
Purpose of the Study:
- To review current viral vector technologies for gene delivery to the central nervous system (CNS).
- To discuss gene therapy strategies for Parkinson's disease, including those targeting motor symptoms and disease modification.
Main Methods:
- Review of existing literature on viral vectors and gene therapy clinical trials for PD.
- Analysis of gene therapy approaches targeting neuronal pathways in the basal ganglia.
Main Results:
- Most clinical trials for PD gene therapy have utilized viral vectors to deliver transgenes to basal ganglia neurons.
- Initial trials focused on genes aimed at relieving major motor symptoms associated with nigrostriatal degeneration.
Conclusions:
- While current gene therapy approaches show potential for symptom relief in PD, more effective disease-modifying strategies are needed.
- Advances in understanding PD genetics and gene delivery systems offer future opportunities for interventions that could slow or halt disease progression.
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