| Project/Area Number |
22790837
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| Research Category |
Grant-in-Aid for Young Scientists (B)
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| Allocation Type | Single-year Grants |
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| Research Field |
Neurology
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| Research Institution | National Center of Neurology and Psychiatry |
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Principal Investigator |
POPIEL HELENA Akiko 独立行政法人国立精神・神経医療研究センター, 神経研究所疾病研究第四部, 科研費研究員 (40467593)
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| Project Period (FY) |
2010 – 2011
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| Project Status |
Completed (Fiscal Year 2011)
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| Budget Amount *help |
¥3,900,000 (Direct Cost: ¥3,000,000、Indirect Cost: ¥900,000)
Fiscal Year 2011: ¥2,080,000 (Direct Cost: ¥1,600,000、Indirect Cost: ¥480,000)
Fiscal Year 2010: ¥1,820,000 (Direct Cost: ¥1,400,000、Indirect Cost: ¥420,000)
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| Keywords | 脳神経疾患 / 蛋白質 / 薬剤スクリーニング / バイオテクノロジー / オリゴマー |
|---|
| Research Abstract |
The polyglutamine(polyQ) diseases are neurological diseases caused by the accumulation of proteins in the brain. Recent studies show that protein complexes named oligomers that are formed prior to their accumulation are the toxic species. Towards establishing a therapy against the polyQ diseases, we screened for chemicals that specifically bind to the disease-causing protein of the polyQ diseases and also inhibit oligomer formation, using the new technologies SPR and FCS. We found that QSI-1, which exerted effects in vitro and in cell culture also exerted therapeutic effects against the neurological phenotypes and protein accumulation of a polyQ disease fly model. Furthermore, administration of QSI-1 to polyQ disease mouse models resulted in amelioration of the neurological symptoms. Our results demonstrate a great advancement towards establishing a therapy for the polyQ diseases. Furthermore, our strategy of screening for therapeutic candidates can be utilized for other neurological diseases that are caused by oligomer formation.
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