Short interfering nucleic acid (siNA) molecules containing a 2′ internucleoside linkage (3dT)
申请人:SIRNA THERAPEUTICS, INC.
公开号:US10738308B2
公开(公告)日:2020-08-11
The present invention relates to RNAi molecules, and compositions thereof, comprising a 2′ internucleoside linkage connecting the nucleotide at position 1 and the nucleotide at position 2 at the 5′ end of the antisense strand. Specifically, the invention relates to single- and double-stranded short interfering nucleic acid (siNA) molecules that are capable of mediating RNA interference comprising 5′ modified nucleotides that comprise, among other potential modifications, a 2′ internucleoside linkage. The invention further relates to 5′ modified nucleotides used as reagents to generate the RNAi molecules of the invention and methods of using the disclosed RNAi molecules.
本发明涉及RNAi分子及其组合物,其包含连接反义链5′端的1位核苷酸和2位核苷酸的2′核苷间连接。具体地说,本发明涉及能够介导 RNA 干扰的单链和双链短干扰核酸(siNA)分子,这些分子包含 5′修饰的核苷酸,这些核苷酸除其他可能的修饰外,还包含 2′核苷间连接。本发明进一步涉及用作试剂生成本发明 RNAi 分子的 5′修饰核苷酸以及使用所公开 RNAi 分子的方法。