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OLIGONUCLEOTIDES INHIBITEURS ET LEUR UTILISATION EN THERAPIE
专利权人:
SAREPTA THERAPEUTICS; INC.
发明人:
UHLMANN, EUGEN,JURK, MARION,LEHMANN, THOMAS
申请号:
CA2897125
公开号:
CA2897125A1
申请日:
2014.01.13
申请国别(地区):
CA
年份:
2014
代理人:
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.
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