Exploring the Effect of Sequence Length and Composition on Allele-Selective Inhibition of Human Huntingtin Expression by Single-Stranded Silencing RNAs

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 therapeu...

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Detalhes bibliográficos
Main Authors: 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.
Formato: Artigo
Idioma:Inglês
Publicado em: Mary Ann Liebert, Inc. 2014
Assuntos:
Original Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4026301/
https://ncbi.nlm.nih.gov/pubmed/24694346
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/nat.2013.0476
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