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Article

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

Biological chemistry - 1 Apr 2011

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

Abstract excerpt

Spinocerebellar ataxia-3 (also known as Machado-Joseph disease) is an incurable neurodegenerative disorder caused by expression of a mutant variant of ataxin-3 (ATX3) protein. Inhibiting expression of ATX3 would provide a therapeutic strategy, but indiscriminant inhibition of both wild-type and mutant ATX3 might lead to undesirable side effects. An ideal silencing agent would block expression of mutant ATX3 while...

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Identifiers

PMID: 21294677
DOI: 10.1515/BC.2011.045
PMCID: PMC3908450

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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