2010 Fiscal Year Final Research Report
Establishment of a novel RNAi knockdown targeting nucleotide variations in neurodegenerative disease-causing alleles.
| Project/Area Number |
20390251
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| Research Category |
Grant-in-Aid for Scientific Research (B)
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| Allocation Type | Single-year Grants |
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| Section | 一般 |
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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 |
HOHJOH Hirohiko National Center of Neurology and Psychiatry, 神経研究所神経薬理研究部, 室長 (60238722)
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| Project Period (FY) |
2008 – 2010
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| Keywords | 臨床神経分子遺伝学 / RNAi |
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| Research Abstract |
Allele-specific gene silencing by RNAi (allele-specific RNAi : ASP-RNAi) is an advanced application of RNAi techniques, by which the expression of an allele of interest can be specifically inhibited. Thus, ASP-RNAi is therapeutically useful for specifically inhibiting the expression of disease-causing alleles without suppressing the expression of corresponding wild-type alleles. To achieve ASP-RNAi, it is vital to design small interfering RNAs (siRNAs) conferring allele discrimination. We developed an easy assay system with reporter alleles. Using our assay system, we successfully designed and selected competent siRNAs conferring ASP-RNAi against mutant Prion Protein (PRNP) and Huntingtin (HTT) genes.
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[Journal Article] Three human ARX mutations cause the lissencephaly-like and mental retardation with epilepsy-like pleiotropic phenotypes in mice2009
Author(s)
Kitamura K., Itou Y., Yanazawa M., Ohsawa M., Suzuki-Migishima R., Umeki Y., Hohjoh H., Yanagawa Y., Shinba T., Itoh M., Nakamura K., Goto Y.
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Journal Title
Hum.Mol.Genet. 18
Pages: 3708-3724
Peer Reviewed
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[Remarks] ホームページ等
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