Tailor-made RNAi knockdown against triplet repeat disease-causing alleles
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- Masaki Takahashi
- National Institute of Neuroscience and
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- Shoko Watanabe
- National Institute of Neuroscience and
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- Miho Murata
- National Institute of Neuroscience and
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- Hirokazu Furuya
- Neuro-Muscular Center, National Oomuta Hospital, Fukuoka 837-0911, Japan
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- Ichiro Kanazawa
- National Institute of Neuroscience and
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- Keiji Wada
- National Institute of Neuroscience and
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- Hirohiko Hohjoh
- National Institute of Neuroscience and
書誌事項
- 公開日
- 2010-11-22
- DOI
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- 10.1073/pnas.1012153107
- 公開者
- Proceedings of the National Academy of Sciences
この論文をさがす
説明
<jats:p> 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 <jats:italic>Huntingtin</jats:italic> 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. </jats:p>
収録刊行物
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- Proceedings of the National Academy of Sciences
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Proceedings of the National Academy of Sciences 107 (50), 21731-21736, 2010-11-22
Proceedings of the National Academy of Sciences
