发明名称 INTERFERING RNA DUPLEX HAVING BLUNT-ENDS AND 3'-MODIFICATIONS
摘要 The present invention relates to double-stranded RNA compounds with at least one blunt end comprising at least one 3'-end of Formula (I): wherein X is O or S R1 and R2 are independently OH, NH2, SH, alkyl, aryl, alkyl-aryl, aryl- alkyl, where alkyl, aryl, alkyl-aryl, aryl-alkyl can be substituted by additional heteroatoms and functional groups, preferably a heteroatom select ed from the group of N, O, or S or a functional group selected from the group O H, NH2, SH, carboxylic acid or ester; or R1 and R2 may be of formula Y-Z where Y is O, N, S and Z is H, alkyl, aryl, alkyl-aryl, aryl-alkyl, where alkyl, ary l, alkyl-aryl, aryl-alkyl can be substituted by additional heteroatoms, preferably a heteroatom selected from the group of N, O, or S; and wherein said double-stranded RNA mediates RNA interference. Preferred 3'end modifications are: phosphate, phosphorothioate, abasic ribonucleoside, hydroxyphopyl phosphodiester.
申请公布号 CA2536333(A1) 申请公布日期 2005.03.10
申请号 CA20042536333 申请日期 2004.08.27
申请人 NOVARTIS AG 发明人 NATT, FRANCOIS JEAN-CHARLES;HALL, JONATHAN;WEILER, JAN;BOLOGNA, JEAN-CHARLES;HANER, ROBERT
分类号 C12N15/11;A61K48/00 主分类号 C12N15/11
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