发明名称 DOUBLE STRAND COMPOSITIONS COMPRISING DIFFERENTIALLY MODIFIED STRANDS FOR USE IN GENE MODULATION
摘要 The present invention provides double stranded compositions wherein the first strand is modified to have a particular motif and the second strand is modified a selected motif. The motifs are defined by positioning of differentially modified nucleosides wherein at least the sugar moieties are different. More particularly, the present compositions comprise an antisense strand that is modified to have a positional/full motif and the sense strand is modified to have an alternating motif, a hemimer motif, a blockmer motif, a gapped motif, a positional motif, a positional/full motif or a fully modified motif. Each strand further comprises one or more phosphorothioate internucleoside linkage. The compositions are useful for targeting selected nucleic acid molecules and modulating the expression of one or more genes. In preferred embodiments the compositions of the present invention hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA. The present invention also provides methods for modulating gene expression.
申请公布号 US2015315579(A1) 申请公布日期 2015.11.05
申请号 US201514616595 申请日期 2015.02.06
申请人 ISIS PHARMACEUTICALS, INC. 发明人 Bhat Balkrishen;Prakash Thazha P.;Allerson Charles;Kinberger Garth A.;Marcusson Eric G.;Swayze Eric E.
分类号 C12N15/113 主分类号 C12N15/113
代理机构 代理人
主权项
地址 Carlsbad CA US