Mechanism of allele-selective inhibition of huntingtin expression by duplex RNAs that target CAG repeats: function through the RNAi pathway

@inproceedings{Hu2012MechanismOA,
  title={Mechanism of allele-selective inhibition of huntingtin expression by duplex RNAs that target CAG repeats: function through the RNAi pathway},
  author={Jiaxin Hu and Jing Liu and Dongbo Yu and Yongjun Chu and David R. Corey},
  booktitle={Nucleic acids research},
  year={2012}
}
Huntington's disease is an incurable neurodegenerative disorder caused by expansion of a CAG trinucleotide repeat within one allele of the huntingtin (HTT) gene. Agents that block expression of mutant HTT and preserve expression of wild-type HTT target the cause of the disease and are an alternative for therapy. We have previously demonstrated that mismatch-containing duplex RNAs complementary to the expanded trinucleotide repeat are potent and allele-selective inhibitors of mutant HTT… CONTINUE READING
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Single-stranded RNAs that function through RNAi are potent and allele-selective inhibitors of huntingtin expression

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