发明名称 INHIBITORY OLIGONUCLEOTIDES AND THEIR USE IN THERAPY
摘要 <p>Inhibitory oligonucleotide having the general formula: (I) are disclosed which can be used in pharmaceutical compositions, whereby in formula (I) C is cytidine or a derivative thereof, whereby the cytidine derivative is selected from the group consisting of 5-methylcytidine, a cytidine-like nucleotide having a chemical modification involving the cytosine base, cytidine nucleoside sugar, or both the cytosine base and the cytidine nucleoside sugar, 2'-0-methylcytidine, 5-bromocytidine, 5-hydroxycytidine, ribocytidine and cytosine-P-D-arabinofuranoside, G is guanosine or a derivative thereof, whereby the guanosine derivative is selected from the group consisting of 7-deazaguanosine, a guanosine-like nucleotide having a chemical modification involving the guanine base, the guanosine nucleoside sugar or both the guanine base and the guanosine nucleoside sugar, X1 and X3 is any nucleotide sequence with 0 to 12 bases and each nucleotide is independent of any other, X2 is any nucleotide sequence having 0 to 3 nucleotides, N2 and N3 are each independently any nucleotide, N4 and N7 is a pyrimidine or a modified pyrimidine, N5 is a purin or a modified purin, N6 is a modified pyrimidine, A or a modified purin, wherein at least two of the nucleotides N4, N5, N6 or N7 are modified purins or modified pyrimidines.</p>
申请公布号 CA2897125(A1) 申请公布日期 2014.07.17
申请号 CA20142897125 申请日期 2014.01.13
申请人 SAREPTA THERAPEUTICS, INC. 发明人 UHLMANN, EUGEN;JURK, MARION;LEHMANN, THOMAS
分类号 C12N15/117;A61K31/7088;A61P29/00;A61P37/02;C07H21/00 主分类号 C12N15/117
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