摘要 |
The present invention provides iRNA agents comprising at least one subunit of the formula (I): wherein: A and B are each independently for each occurrence O, N(R N) or S; X and Y are each independently for each occurrence H, OH, a hydroxyl protecting group, a phosphate group, a phosphodiester group, an activated phosphate group, an activated phosphite group, a phosphoramidite, a solid support, -P(Z')(Z")0- nucleoside, -P(Z')(Z")O-oligonucleotide, a lipid, a PEG, a steroid, a lipophile, a polymer, -P(Z')(Z")O-Linker-OP(Z"')(Z"")O-oligonucleotide, a nucleotide, an oligonucleotide, - P(Z')(Z")-formula(I), -P(Z')(Z")-or -Linker-R; R is L G, -Linker-L G, or has the structure shown below: L G is independently for each occurrence a carbohydrate, e.g., monosaccharide, disaccharide, trisaccharide, tetrasaccharide, oligosaccharide, polysaccharide; R N is independently for each occur-remce H, methyl, ethyl, propyl, isopropyl,butyl, or benzyl; and Z', Z", Z"' and Z are each independently for each occurrence O or S. |
申请人 |
ALNYLAM PHARMACEUTICALS, INC. |
发明人 |
MANOHARAN, MUTHIAH;RAJEEV, KALLANTHOTTATHIL G.;NARAYANANNAIR, JAYAPRAKASH K.;MAIER, MARTIN |