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Updated: Jun 13, 2026

Ole Isacson: Development of New Therapies for Parkinson's Disease
Published on: April 29, 2007
Gene therapy for Parkinson's disease
1Department of Neurological Surgery, Okayama University Graduate School of Medicine, 2-5-1, Shikata-cho, Okayama, 700-8558, Japan. tyasu37@cc.okayama-u.ac.jp
Abstract:
Parkinson's disease is characterized by the degeneration of the nigrostriatal dopaminergic neurons with the manifestation of tremor, rigidity, akinesia, and disturbances of postural reflexes. Medication using L-DOPA and surgeries including deep brain stimulation are the established therapies for Parkinson's disease. Cell therapies are also effective and have rapidly developed with the recent advancement in molecular biological technology including gene transfer. In this review, ex vivo gene therapy using genetically engineered cell transplantation for Parkinson's disease model of animals is described, including catecholamine/neurotrophic factor-secreting cell transplantation with or without encapsulation, as well as in vivo gene therapy using direct injection of viral vector to increase dopamine-production, ameliorate the survival of dopaminergic neurons, correct the deteriorated microenvironment, or normalize genetic abnormality. Furthermore, the future directions for clinical application are described together with recent clinical trials of gene therapy.
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