{"@context":{"@vocab":"https://cir.nii.ac.jp/schema/1.0/","rdfs":"http://www.w3.org/2000/01/rdf-schema#","dc":"http://purl.org/dc/elements/1.1/","dcterms":"http://purl.org/dc/terms/","foaf":"http://xmlns.com/foaf/0.1/","prism":"http://prismstandard.org/namespaces/basic/2.0/","cinii":"http://ci.nii.ac.jp/ns/1.0/","datacite":"https://schema.datacite.org/meta/kernel-4/","ndl":"http://ndl.go.jp/dcndl/terms/","jpcoar":"https://github.com/JPCOAR/schema/blob/master/2.0/"},"@id":"https://cir.nii.ac.jp/crid/1363670319043136896.json","@type":"Article","productIdentifier":[{"identifier":{"@type":"DOI","@value":"10.1073/pnas.1012153107"}},{"identifier":{"@type":"URI","@value":"https://pnas.org/doi/pdf/10.1073/pnas.1012153107"}},{"identifier":{"@type":"PMID","@value":"21098280"}}],"dc:title":[{"@value":"Tailor-made RNAi knockdown against triplet repeat disease-causing alleles"}],"description":[{"type":"abstract","notation":[{"@value":"<jats:p>\n            Nucleotide variations, including SNPs, in the coding regions of disease genes are important targets for RNAi treatment, which is a promising medical treatment for intractable diseases such as triplet repeat diseases. However, the identification of such nucleotide variations and the design of siRNAs conferring disease allele-specific RNAi are quite difficult. In this study we developed a pull-down method to rapidly identify coding SNP (cSNP) haplotypes of triple repeat, disease-causing alleles, and we demonstrated disease allele-specific RNAi that targeted cSNP sites in mutant\n            <jats:italic>Huntingtin</jats:italic>\n            alleles, each of which possessed a different cSNP haplotype. Therefore, the methods presented here allow for allele-specific RNAi knockdown against disease-causing alleles by using siRNAs specific to disease-linked cSNP haplotypes, and advanced progress toward tailor-made RNAi treatments for triplet repeat diseases.\n          </jats:p>"}]}],"creator":[{"@id":"https://cir.nii.ac.jp/crid/1030018198853554688","@type":"Researcher","personIdentifier":[{"@type":"KAKEN_RESEARCHERS","@value":"00549529"},{"@type":"NRID","@value":"1000000549529"},{"@type":"NRID","@value":"9000005781435"},{"@type":"NRID","@value":"9000256850095"},{"@type":"NRID","@value":"9000256308060"},{"@type":"NRID","@value":"9000256850365"},{"@type":"NRID","@value":"9000020215634"},{"@type":"RESEARCHMAP","@value":"https://researchmap.jp/tk_masaki"}],"foaf:name":[{"@value":"Masaki Takahashi"}],"jpcoar:affiliationName":[{"@value":"National Institute of Neuroscience and"}]},{"@id":"https://cir.nii.ac.jp/crid/1380579815662991875","@type":"Researcher","foaf:name":[{"@value":"Shoko Watanabe"}],"jpcoar:affiliationName":[{"@value":"National Institute of Neuroscience and"}]},{"@id":"https://cir.nii.ac.jp/crid/1380579815662991878","@type":"Researcher","foaf:name":[{"@value":"Miho Murata"}],"jpcoar:affiliationName":[{"@value":"National Institute of Neuroscience and"},{"@value":"National Center Hospital, National Center of Neurology and Psychiatry, Tokyo 187-8502, Japan; and"}]},{"@id":"https://cir.nii.ac.jp/crid/1380579815662991877","@type":"Researcher","foaf:name":[{"@value":"Hirokazu Furuya"}],"jpcoar:affiliationName":[{"@value":"Neuro-Muscular Center, National Oomuta Hospital, Fukuoka 837-0911, Japan"}]},{"@id":"https://cir.nii.ac.jp/crid/1380579815662991876","@type":"Researcher","foaf:name":[{"@value":"Ichiro Kanazawa"}],"jpcoar:affiliationName":[{"@value":"National Institute of Neuroscience and"},{"@value":"National Center Hospital, National Center of Neurology and Psychiatry, Tokyo 187-8502, Japan; and"}]},{"@id":"https://cir.nii.ac.jp/crid/1380579815662991874","@type":"Researcher","foaf:name":[{"@value":"Keiji Wada"}],"jpcoar:affiliationName":[{"@value":"National Institute of Neuroscience and"}]},{"@id":"https://cir.nii.ac.jp/crid/1380579815662991873","@type":"Researcher","foaf:name":[{"@value":"Hirohiko Hohjoh"}],"jpcoar:affiliationName":[{"@value":"National Institute of Neuroscience and"}]}],"publication":{"publicationIdentifier":[{"@type":"PISSN","@value":"00278424"},{"@type":"EISSN","@value":"10916490"}],"prism:publicationName":[{"@value":"Proceedings of the National Academy of Sciences"}],"dc:publisher":[{"@value":"Proceedings of the National Academy of Sciences"}],"prism:publicationDate":"2010-11-22","prism:volume":"107","prism:number":"50","prism:startingPage":"21731","prism:endingPage":"21736"},"reviewed":"false","dcterms:accessRights":"http://purl.org/coar/access_right/c_abf2","url":[{"@id":"https://pnas.org/doi/pdf/10.1073/pnas.1012153107"}],"createdAt":"2010-11-23","modifiedAt":"2022-04-13","foaf:topic":[{"@id":"https://cir.nii.ac.jp/all?q=Huntingtin%20Protein","dc:title":"Huntingtin Protein"},{"@id":"https://cir.nii.ac.jp/all?q=Nuclear%20Proteins","dc:title":"Nuclear Proteins"},{"@id":"https://cir.nii.ac.jp/all?q=Nerve%20Tissue%20Proteins","dc:title":"Nerve Tissue Proteins"},{"@id":"https://cir.nii.ac.jp/all?q=Polymorphism,%20Single%20Nucleotide","dc:title":"Polymorphism, Single Nucleotide"},{"@id":"https://cir.nii.ac.jp/all?q=HEK293%20Cells","dc:title":"HEK293 Cells"},{"@id":"https://cir.nii.ac.jp/all?q=Haplotypes","dc:title":"Haplotypes"},{"@id":"https://cir.nii.ac.jp/all?q=Trinucleotide%20Repeats","dc:title":"Trinucleotide Repeats"},{"@id":"https://cir.nii.ac.jp/all?q=Gene%20Knockdown%20Techniques","dc:title":"Gene Knockdown Techniques"},{"@id":"https://cir.nii.ac.jp/all?q=Humans","dc:title":"Humans"},{"@id":"https://cir.nii.ac.jp/all?q=Genetic%20Predisposition%20to%20Disease","dc:title":"Genetic Predisposition to Disease"},{"@id":"https://cir.nii.ac.jp/all?q=RNA%20Interference","dc:title":"RNA Interference"},{"@id":"https://cir.nii.ac.jp/all?q=RNA,%20Small%20Interfering","dc:title":"RNA, Small Interfering"},{"@id":"https://cir.nii.ac.jp/all?q=Alleles","dc:title":"Alleles"},{"@id":"https://cir.nii.ac.jp/all?q=HeLa%20Cells","dc:title":"HeLa Cells"}],"relatedProduct":[{"@id":"https://cir.nii.ac.jp/crid/1360283691080037888","@type":"Article","resourceType":"学術雑誌論文(journal article)","relationType":["isReferencedBy"],"jpcoar:relatedTitle":[{"@value":"Supplemental Treatment for Huntington’s Disease with miR-132 that Is Deficient in Huntington’s Disease 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