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Double stranded rna compounds to rhoa and use thereof
专利权人:
QUARK PHARMACEUTICALS INC.
发明人:
FEINSTEIN ELENA,KALINSKI HAGAR,AVKIN-NACHUM SHARON,METT IGOR
申请号:
NZ60409411
公开号:
NZ604094A
申请日:
2011.06.23
申请国别(地区):
NZ
年份:
2014
代理人:
摘要:
Disclosed is a double-stranded nucleic acid molecule (RHOA_48) having the structure: 5&rsquo UGUAGCAAGAUGACUUCUG -Z 3&rsquo (antisense SEQ ID NO:113) 3&rsquo Z&rsquo-ACAUCGUUCUACUGAAGAC -z&rdquo 5&rsquo(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&rsquo-5&rsquo linked ribonucleotide is present in at least one of positions 5, 6, 7 or 8 (5&rsquo>3&rsquo), 2&rsquo-O-Methyl sugar-modified ribonucleotides are present at positions 1, 3, 11, 14, 15, 17 and 18 (5&rsquo>3&rsquo), and Z is a C3Pi-C3OH moiety or a C3Pi-C3Pi moiety covalently attached at the 3&rsquo terminus of the antisense strand wherein on the sense strand a 2&rsquo-O-Methyl sugar-modified ribonucleotide is present at position 1, 2&rsquo-5&rsquo linked ribonucleotides are present at positions 15, 16, 17, 18 and 19 (5&rsquo>3&rsquo) and Z&rsquo is 1-5 consecutive nucleotides or non-nucleotide or unconventional moieties or a combination thereof covalently attached at the 3&rsquo terminus of the sense strand wherein z&rdquo is a capping moiety covalently attached at the 5&rsquo terminus of the sense strand or a pharmaceutically acceptable salt of such nucleic acid molecule.
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