Allele-Selective Inhibition of Mutant Atrophin-1 Expression by Duplex and Single-Stranded RNAs

[Image: see text] Dentatorubral-pallidoluysian atrophy (DRPLA) is a progressive neurodegenerative disorder that currently has no curative treatments. DRPLA is caused by an expansion of a CAG trinucleotide repeat region within the protein-encoding sequence of the atrophin-1 (ATN-1) gene. Inhibition o...

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Autori principali: Hu, Jiaxin, Liu, Jing, Narayanannair, K. Jayaprakash, Lackey, Jeremy G., Kuchimanchi, Satya, Rajeev, Kallanthottathil G., Manoharan, Muthiah, Swayze, Eric E., Lima, Walt F., Prakash, Thazha P., Xiang, Qin, Martinez, Carlos, Corey, David R.
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Chemical Society 2014
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC4108179/
https://ncbi.nlm.nih.gov/pubmed/24981774
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1021/bi500610r
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