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Article

ss-siRNAs allele selectively inhibit ataxin-3 expression: multiple mechanisms for an alternative gene silencing strategy.

Nucleic acids research - 1 Nov 2013

Liu Jing, Yu Dongbo, Aiba Yuichiro, Pendergraff Hannah, Swayze Eric E, Lima Walt F, Hu Jiaxin, Prakash Thazha P, Corey David R

Abstract excerpt

Single-stranded silencing RNAs (ss-siRNAs) provide an alternative approach to gene silencing. ss-siRNAs combine the simplicity and favorable biodistribution of antisense oligonucleotides with robust silencing through RNA interference (RNAi). Previous studies reported potent and allele-selective inhibition of human huntingtin expression by ss-siRNAs that target the expanded CAG repeats within the mutant allele....

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PMID: 23935115
DOI: 10.1093/nar/gkt693
PMCID: PMC3814390

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