2009 Fiscal Year Final Research Report
Molecular therapy for the polyglutamine diseases using a novel brain delivery method of aggregate inhibitor peptides
| Project/Area Number |
20700336
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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 |
Neurochemistry/Neuropharmacology
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| Research Institution | National Center of Neurology and Psychiatry |
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Principal Investigator |
POPIE HELENA Akiko National Center of Neurology and Psychiatry, 神経研究所 疾病研究第四部, 流動研究員 (40467593)
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| Project Period (FY) |
2008 – 2009
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| Keywords | 神経科学 / 脳神経疾患 / ドラッグデリバリー / 血液脳関門 / 創薬 / 神経変性疾患 / ポリグルタミン病 / 凝集阻害 |
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| Research Abstract |
The polyglutamine (PolyQ) diseases are neurological diseases caused by protein aggregation in the brain. We previously identified QBP1, a molecule that inhibits PolyQ disease aggregation. However, for actual use of QBP1 as a therapy, its inefficient delivery to the brain was a problem. In this study, to improve brain delivery of QBP1, we (1) screened for PTDs, which are sequences that allow the delivery of various molecules into cells, that enable brain delivery of QBP1, and (2) to decrease the size of QBP1, we analyzed which part of QBP1 is required for its activity. As a result, we (1) found that fusion of some PTDs to QBP1 appears to enhance its brain delivery, and (2) determined the part of QBP1 required for its therapeutic effect.
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Research Products
(20 results)
