Modified polynucleotides for use in rna interference

作者: Devin Leake , William Marshall , Yurity Fedorov , Anastasia Khvorova , Kimberly Nichols

DOI:

关键词: PolynucleotideNucleotideBiochemistrySmall interfering RNAConjugateCombinatorial chemistryRNA interferenceTerminal (electronics)Sense (molecular biology)Biology

摘要: Methods and compositions for performing RNA interference comprising a wide variety of stabilized siRNAs suitable use in serum-containing media vivo applications, such as therapeutic are provided. These permit effective efficient applications to diagnostics therapeutics through the one or more modifications including orthoesters, terminal conjugates, modified linkages 2'modified nucleotides. Uniquely have been developed that reduces off-target effects incurred gene-silencing. The include phosphorylation first 5' antisense nucleotide; 2' carbon second first, second, third nucleotides; optionally sense nucleotide. Control exaequo molecules also siRNA related control, trackability agents with specific stability were developed.

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