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    • 81. 发明专利
    • Double stranded rna compounds to rhoa and use thereof
    • NZ604094A
    • 2014-11-28
    • NZ60409411
    • 2011-06-23
    • QUARK PHARMACEUTICALS INC
    • FEINSTEIN ELENAKALINSKI HAGARAVKIN-NACHUM SHARONMETT IGOR
    • C12N15/113A61K31/713
    • Disclosed is a double-stranded nucleic acid molecule (RHOA_48) having the structure: 5’ UGUAGCAAGAUGACUUCUG -Z 3’ (antisense SEQ ID NO:113) 3’ Z’-ACAUCGUUCUACUGAAGAC -z” 5’(sense SEQ ID NO:79) wherein each of A, C, G and U is independently an unmodified ribonucleotide, a modified ribonucleotide, or an unconventional moiety; wherein on the antisense strand a mirror nucleotide or a 2’-5’ linked ribonucleotide is present in at least one of positions 5, 6, 7 or 8 (5’>3’), 2’-O-Methyl sugar-modified ribonucleotides are present at positions 1, 3, 11, 14, 15, 17 and 18 (5’>3’), and Z is a C3Pi-C3OH moiety or a C3Pi-C3Pi moiety covalently attached at the 3’ terminus of the antisense strand; wherein on the sense strand a 2’-O-Methyl sugar-modified ribonucleotide is present at position 1, 2’-5’ linked ribonucleotides are present at positions 15, 16, 17, 18 and 19 (5’>3’); and Z’ is 1-5 consecutive nucleotides or non-nucleotide or unconventional moieties or a combination thereof covalently attached at the 3’ terminus of the sense strand; wherein z” is a capping moiety covalently attached at the 5’ terminus of the sense strand; or a pharmaceutically acceptable salt of such nucleic acid molecule.