Article
Exploring the effect of sequence length and composition on allele-selective inhibition of human huntingtin expression by single-stranded silencing RNAs.
Nucleic acid therapeutics - 1 Jun 2014
Hu Jiaxin, Liu Jing, Yu Dongbo, Aiba Yuichiro, Lee Suheung, Pendergraff Hannah, Boubaker Jihane, Artates Jonathan W, Lagier-Tourenne Clotilde, Lima Walt F, Swayze Eric E, Prakash Thazha P, Corey David R
Abstract excerpt
Mutant huntingtin (HTT) protein is the cause of Huntington's disease (HD), an incurable neurological disorder. Almost all patients are heterozygous for mutant HTT and approaches that reduce levels of mutant HTT while leaving expression of wild-type HTT intact might be ideal options for therapeutic development. We have developed several allele-selective strategies for silencing HTT, including single-stranded...
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