Establishment of tailor-made RNAi knockdown technique against triplet repeat disease-causing alleles

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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.