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Allele-selective inhibition of ataxin-3 (ATX3) expression by antisense oligomers and duplex RNAs

Spinocerebellar ataxia-3 (SCA3) (also known as Machado Joseph Disease) is an incurable neurodegenerative disorder caused by expression of a mutant variant of ataxin-3 protein (ATX3). Inhibiting expression of ATX-3 would provide a therapeutic strategy, but indiscriminant inhibition of both wild-type...

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Bibliografiske detaljer
Main Authors: Hu, Jiaxin, Gagnon, Keith T., Liu, Jing, Watts, Jonathan K., Syeda-Nawaz, Jeja, Bennett, C. Frank, Swayze, Eric E., Randolph, John, Chattopadhyaya, Jyoti, Corey, David R.
Format: Artigo
Sprog:Inglês
Udgivet: 2011
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3908450/
https://ncbi.nlm.nih.gov/pubmed/21294677
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1515/BC.2011.045
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