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- W2068964702 abstract "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 the wild type, Q22G (5.9%). Furthermore, unexpectedly, the expression of another wild-type allele, Q22C, was also much less suppressed (22.5%) by this siRNA possibly due to difference of the secondary structure of the target RNA. This is the first report of sequence-independent discrimination of mutant and wild-type alleles by siRNA. Ann Neurol 2004;56:124–129" @default.
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- W2068964702 date "2004-01-01" @default.
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- W2068964702 title "Sequence-dependent and independent inhibition specific for mutant ataxin-3 by small interfering RNA" @default.
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- W2068964702 doi "https://doi.org/10.1002/ana.20141" @default.
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