2010 Fiscal Year Final Research Report
Establishment of tailor-made RNAi knockdown technique against triplet repeat disease-causing alleles
| Project/Area Number |
21890312
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| Research Category |
Grant-in-Aid for Research Activity Start-up
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| Allocation Type | Single-year Grants |
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| Research Field |
General medical chemistry
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| Research Institution | National Center of Neurology and Psychiatry |
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Principal Investigator |
TAKAHASHI Masaki 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・神経薬理研究部, 流動研究員 (00549529)
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| Project Period (FY) |
2009 – 2010
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| Keywords | 発現制御 / RNAi治療 / SNP / 脳神経疾患 / 対立遺伝子 / トリプレットリピート病 |
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| Research Abstract |
We present determination of the coding SNP (cSNP) haplotypes of triplet repeat disease-causing alleles by a novel pull-down method, and demonstrate disease-allele specific RNAi targeting such cSNP sites in the mutant Huntingtin alleles, each of which possesses a different cSNP haplotype. Thus, our procedures allow for allele-specific RNAi against disease-causing alleles by using appropriate siRNAs as the disease-linked cSNP haplotype demands. Therefore, our current progress paves the way to the achievement of a tailor-made RNAi treatment for triplet repeat diseases and other diseases using cSNPs as RNAi targets.
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