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03-07-2013, 10:50 PM | #1 | |||
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Raising an Enzyme's Levels May Help Treat Parkinson's Disease
- Feb 22 2013 http://www.pdf.org/en/science_news/r.../pr_1361552717 Increasing the amount of glucocerebrosidase (GBA enzyme) in the brain may help to treat Parkinson's disease ... may also help keep harmful deposits of the protein alpha-synuclein from accumulating in the brain. Previous observations suggested a link between a deficiency in the GBA enzyme and PD. For example, people with PD typically have lower amounts of GBA enzyme in their brains. Some people living with PD also have mutations in the GBA1 gene, which makes this enzyme and causes Gaucher's, a disease which can include PD-like symptoms... ... They studied the concept, using two mouse models: mice with symptoms of Gaucher's disease and other mice with PD-like symptoms. Researchers treated mice with gene therapy - an injection of healthy copies of the GBA1 gene... Results Six months after the gene therapy treatment, the mice with symptom's of Gaucher's disease, had reduced fatty deposits in the brain and fewer clumps of misfolded alpha-synuclein and other proteins associated with nuerological disorders. The treatment also restored the impaired memory of these mice to normal levels. Like people with PD, mice treated to have PD-like symptoms had lower amounts of active GBA enzyme than normal mice. Mice with the highest levels of alpha-synuclein had the lowest levels of GBA enzyme. In the PD mice, the gene therapy treatment decreased the amount of free alpha-synuclein in the brain, but not of alpha-synuclein clumps. What Does It Mean? This study adds to existing evidence that a deficiency in the GBA enzyme may contribute to PD symptoms. It also suggest that fixing this deficiency could be therapeutic... ...It is possible that the enzyme normally clears out excess alpha-synuclein, that it helps the protein fold properly to prevent its clumping together, or that is breaks up protein clumps. Further studies are needed to determine the precise role of the enzyme in PD... Reference: Sardi SP, Clarke J, Viel C, Chan M, Tamsett TJ, Treleaven CM, Bu J, Sweet L, Passini MA, Dodge JC, Yu WH, Sidman RL, Cheng SH, Shihabuddin LS (2013) Augmenting CNS glucocerebrosidase activity as a therapeutic strategy for parkinsonism and other Gaucher-related synucleinopathies. Proceedings of the National Academy of Sciences:1-6.
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