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US09574191B2 Selective inhibition of polyglutamine protein expression 有权
选择性抑制聚谷氨酰胺蛋白表达

Selective inhibition of polyglutamine protein expression
摘要:
The present invention relates to the selective inhibition of protein expression of CAG repeat-related disease proteins such as Huntingtin Disease Protein and Ataxin-3 using double-stranded RNAs and nucleic acid analogs. Chemically-modified RNAs having at least one mismatch as compared to the target CAG repeat sequence are specifically contemplated.
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