Parkinson's Disease Basic Science
Parkinson's disease (PD) is caused by progressive cell death in a small part of the brain that helps control movements by releasing the chemical dopamine. Levodopa is the mainstay of PD therapy, but must be converted in the brain into dopamine by an enzyme called AADC (aromatic L-amino acid decarboxylase). Researchers in California presented results of a long-term gene therapy trial in monkeys with a condition that mimics PD. They introduced the gene for AADC into the affected parts of the brains of four monkeys, using a virus as a carrier. After three-and-a-half years, the gene was still making the enzyme widely throughout the affected part of the brain, and the monkeys required much less levodopa to control their PD symptoms. Importantly, the dose of levodopa could be lowered to the point that it no longer caused uncontrolled movements called dyskinesias, which are a major complication of late-stage PD treatment. These promising results suggest that this approach may work in PD patients as well. [S28.004]
Parkinson's Disease Genetics
Most cases of PD are thought to result from a combination of genes and environmental factors such as pesticides and other poisons. Genes that cause PD by themselves are rare. An exception is found in a pair of families from Greece and Italy, who carry a mutation in a gene for a brain protein called alpha-synuclein. While this mutation is exceedingly rare, it has already taught PD researchers much about the dise
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Contact: Kathy Stone
kstone@aan.com
651-695-2763
American Academy of Neurology
4-May-2004