Parkin May Remove the Disease in Parkinson's Disease

Researchers studying a form of inherited Parkinson's disease have identified a gene that when overexpressed produces a protein that protects neurons from the degenerative effects of the disease.

Investigators at the Ecole Polytechnique Federale de Lausanne (Switzerland) worked with a rat model that mimicked familial Parkinson's disease. They found that mutations in two genes, alpha-synuclein and parkin, were present in most cases of familial Parkinson's disease.

To study the role of these genes in the disease process, they injected rats sub-cranially with lentiviral vectors containing the genes for normal parkin as well as the mutated form of alpha-synuclein. A control group was injected with only the alpha-synuclein gene. Results published in the December 2, 2004, online edition of the Proceedings of the [U.S.] National Academy of Sciences showed that the animals receiving both genes maintained normal neuron activity while those receiving only alpha-synuclein displayed symptoms of neural degeneration. Furthermore, rats that had been injected with parkin showed an increase in Lewy body-type intracellular deposits. This increase suggests that parkin helps to sequester toxic substances in the insoluble Lewy bodies.

Senior author Dr. Patrick Aebischer, president of the Ecole Polytechnique Federale de Lausanne, said, "These observations could translate into the clinic either through a direct gene therapy approach or through the identification of small drugs which enhance the expression of the endogenous parkin.”



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