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Chimeric single-stranded antisense polynucleotides and double-stranded antisense agent
Chimeric single-stranded antisense polynucleotides and double-stranded antisense agent
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机译:嵌合单链反义多核苷酸和双链反义剂
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摘要
Chimeric single-stranded polynucleotides and double-stranded antisense agents useful for modifying the expression of a target gene by means of an antisense effect are disclosed. The chimeric single-stranded antisense polynucleotide and double-stranded antisense agents comprise a central nucleotide region flanked by a first 5'-wing region and a first 3'-wing region of modified nucleotides, which are themselves flanked by a second 5'-wing region and/or a second 3'-wing region of nucleotides that have a low affinity for proteins and/or that have higher resistance to DNase or RNase than a natural DNA or RNA and are missing in a cell when the chimeric polynucleotide delivered. The double-stranded antisense agent further comprises a complementary strand annealed to the antisense strand. The polynucleotide can be used to modify RNA transcription levels, miRNA activity, or protein levels in cells.
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