Sequence-dependent and independent inhibition specific for mutant ataxin-3 by small interfering RNA
Sequence-dependent and independent inhibition specific for mutant ataxin-3 by small interfering RNA
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DOI：
10.1002/ana.20141
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发表时间：
2004-07-01
影响因子：
11.2
通讯作者：
Mizusawa, H
中科院分区：
文献类型：
--
作者：
Li, Y;Yokota, T;Mizusawa, H
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.
