具高活性及減低脫靶之siRNA構造农业专利-农业学术服务平台

发明人:: MINOMI, KENJIROU,味吞宪二郎,味吞憲二郎,HARBORTH, JENS,贺柏士　詹斯,賀柏士　詹斯,CINA, CIMA,希纳　希玛,希納　希瑪,YING, WENBIN,尹　温平,尹　溫平,ZHENG, JANE,郑雪英,VAISH, NARENDRA,瓦许　纳瑞达,瓦許　納瑞達,贺柏士詹斯,賀柏士詹斯,希纳希玛,希納希瑪,尹温平,尹溫平,瓦许纳瑞达,瓦許納瑞達

摘要：: This invention provides compounds, compositions and methods for modulating the expression of target genes using RNA interference. RNAi structures and molecules of this invention can be used for modulating or silencing the expression of genes, with high levels of RNAi activity and reduced off target actions. Advantageous structures include siRNAs targeted to any gene having one or more 2'-deoxy nucleotides located in the seed region. The RNA interference molecules can be used in methods for preventing or treating diseases.本發明提供用於使用RNA干擾來調節靶基因之表現之化合物、組合物及方法。本發明之RNAi構造及分子可用於以高RNAi活性程度及減低脫靶作用來調節或靜默基因表現。有利構造包含靶向任一具有一或多個位於種子區(seed region)中之2'-去氧核苷酸之基因之siRNA。該等RNA干擾分子可用於預防或治療疾病之方法中。