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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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| Main Authors: | , , , , , , , , , |
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| Format: | Artigo |
| Sprog: | Inglês |
| Udgivet: |
2011
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| 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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