The inventors have examined the means for providing more efficacious gene expression blocking compounds. The inventors have discovered new structural features that surprisingly improve the efficacy of gene expression blocking molecules. These features include the presence of multiple 3' ends and a linker at the 5' ends. Surprisingly, these features improve the efficacy of the gene expression blocking compounds in a manner that decreases the compound's biologic instability. Even more surprisingly, this effect has been found to be applicable to both DNA and RNA oligonucleotide-based compounds and to have application in traditional antisense and RNAi technologies.
本发明者研究了提供更有效
基因表达阻断化合物的方法。本发明者发现了新的结构特征,这些特征令人惊讶地提高了
基因表达阻断分子的功效。这些特征包括多个 3'末端和 5'末端的连接体。令人惊讶的是,这些特征以降低化合物
生物不稳定性的方式提高了
基因表达阻断化合物的功效。更令人惊讶的是,这种效果已被发现适用于 DNA 和 RNA 寡核苷酸化合物,并可应用于传统的反义和 RNAi 技术。