Single-Stranded RNAs Use RNAi to Potently and Allele-Selectively Inhibit Mutant Huntingtin Expression

Mutant huntingtin (HTT) protein causes Huntington’s Disease (HD), an incurable neurological disorder. Silencing mutant HTT using nucleic acids would eliminate the root cause of HD. Developing nucleic acid drugs is challenging, and an ideal clinical approach to gene silencing would combine the simpli...

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Autores principales: Yu, Dongbo, Pendergraff, Hannah, Liu, Jing, Kordasiewicz, Holly B., Cleveland, Don W., Swayze, Eric E., Lima, Walt F., Crooke, Stanley T., Prakash, Thazha P., Corey, David R.
Formato: Artigo
Lenguaje:Inglês
Publicado: 2012
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Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC3444165/
https://ncbi.nlm.nih.gov/pubmed/22939619
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.cell.2012.08.002
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