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Article

Sequence-dependent and independent inhibition specific for mutant ataxin-3 by small interfering RNA.

Annals of neurology - 1 Jul 2004

Li Yi, Yokota Takanori, Matsumura Ryusuke, Taira Kazunari, Mizusawa Hidehiro

Abstract excerpt

In Machado-Joseph disease (MJD) gene, there is a C/G polymorphism immediately after the CAG repeat; the expanded CAG repeat tract is exclusively followed by C, whereas about half of wild-type alleles are followed by G. Using this C/G polymorphism, we have engineered the small interfering RNA (siRNA) which decreased the expression of mutant ataxin-3, Q79C, by 96.0%, whereas there was minimal reduction on that of...

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PMID: 15236410
DOI: 10.1002/ana.20141

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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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