发明名称 INHIBITORY OLIGONUCLEOTIDES AND THEIR USE IN THERAPY.
摘要 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.
申请公布号 MX2015009006(A) 申请公布日期 2016.02.11
申请号 MX20150009006 申请日期 2014.01.13
申请人 SAREPTA THERAPEUTICS, INC. 发明人 EUGEN UHLMANN;THOMAS LEHMANN;MARION JURK
分类号 C12N15/117;A61K31/7088;A61P29/00;A61P37/02;C07H21/00 主分类号 C12N15/117
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