Article
Allele-selective inhibition of huntingtin expression by switching to an miRNA-like RNAi mechanism.
Chemistry & biology - 24 Nov 2010
Hu Jiaxin, Liu Jing, Corey David R
Abstract excerpt
Inhibiting expression of huntingtin (HTT) protein is a promising strategy for treating Huntington's disease (HD), but indiscriminant inhibition of both wild-type and mutant alleles may lead to toxicity. An ideal silencing agent would block expression of mutant HTT while leaving expression of wild...
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