Development of a novel approach toward nucleic acids-based therapeutics

点击翻译按钮获取中文摘要

In this work, we aimed to prepare novel chemically modified RNA molecules possessing nuclease resistance and hybridization ability. In addition, we expected that these molecules exhibit editing ability of splicing abnormality. As a result, the resulting hybrid type of 4’-thionucleic acids showed preferable properties concerning of nuclease resistance and hybridization ability. Among the hybrid type of 4’-thionucleic acids developed, 2’-F-2’-thioRNA showed U1i activity arising from recruitment of U1 snRNA on pre-mRNA. Toward the end goal, we investigated the preparation of head-to-head oligonucleotides.