Gene therapy for Parkinson's disease.
Gene therapy for Parkinson's disease.
Where did the research take place?
The study site has not been established. Author addresses may differ from where the research occurred.
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Original abstract
Clinical studies of gene therapy for Parkinson's disease are based on three kinds of strategies. 1. Restoration of dopamine production in the putamen by introducing genes of dopamine-synthesizing enzymes. 2. Protection of nigrostriatal projections by gene transfer of neurotrophic factors into the putamen and substantia nigra. 3. Modulation of subthalamic nucleus neural activity by gene delivery of an enzyme to synthesize inhibitory transmitter y-aminobutyric acid. In all studies, procedures were well tolerated and no adverse effects attributed to viral vectors were reported. The beneficial effects of putaminal gene transfer on motor symptoms have also been confirmed in children with aromatic L-amino acid decarboxylase deficiency. For a neuroprotective strategy, early intervention is necessary before degeneration has proceeded too far.