[EN] LIGAND-MODIFIED DOUBLE-STRANDED NUCLEIC ACIDS<br/>[FR] ACIDES NUCLÉIQUES DOUBLE BRIN MODIFIÉS PAR UN LIGAND
申请人:DICERNA PHARMACEUTICALS INC
公开号:WO2016100401A1
公开(公告)日:2016-06-23
The invention provides for double stranded nucleic acid molecules comprising a 5 'extension of the sense or antisense strand and further comprising a plurality of nucleotides that are conjugated to a ligand and methods of using the double-stranded nucleic acid molecules. Ligand-modified oligomers where the sense stands form a tetraloop provide new potent and stable RNA interference agents. These dsNA molecules are synthesized using a plurality of nucleotides that include ligand-modified monomers, nucleotide analog monomers, modified nucleotide monomers and the like, using standard nucleotide synthetic methods and systems.
该发明提供了包括具有感链或反义链的5'延伸以及进一步包括与配体结合的多个核苷酸的双链核酸分子,以及使用这些双链核酸分子的方法。其中,感链形成四环的配体修饰寡聚物提供了新的强效和稳定的RNA干扰剂。这些双链核酸分子是使用包括配体修饰单体、核苷酸类似物单体、修饰核苷酸单体等的多个核苷酸合成的,使用标准的核苷酸合成方法和系统。