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Tailor-made RNAi knockdown against triplet repeat disease-causing alleles.

Abstract：

: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 Huntingtin 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.

journal_name

Proc Natl Acad Sci U S A

journal_title

Proceedings of the National Academy of Sciences of the United States of America

authors

Takahashi M,Watanabe S,Murata M,Furuya H,Kanazawa I,Wada K,Hohjoh H

doi

10.1073/pnas.1012153107

subject

Has Abstract

pub_date

2010-12-14 00:00:00

pages

21731-6

issue

50

eissn

0027-8424

issn

1091-6490

pii

1012153107

journal_volume

107

pub_type

杂志文章

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