Disclosed herein are double-stranded RNA nucleic acid molecules, which include at least one pyrazolotriazine nucleotide analog and have been modified to exhibit one of the following, increased on-target activity, increased target specificity, enhanced nuclease stability, reduced off target activity and/or reduced immunogenicity when compared to an unmodified or similarly modified dsRNA; pharmaceutical compositions comprising such molecules and methods of use thereof in therapy.
本文公开了双链 RNA 核酸分子,其中包括至少一种
吡唑三嗪核苷酸类似物,与未经修饰或类似修饰的 dsRNA 相比,经过修饰的双链 RNA 核酸分子表现出以下特征之一:靶上活性增强、靶特异性增强、
核酸酶稳定性增强、靶外活性降低和/或免疫原性降低;包含此类分子的药物组合物及其用于治疗的方法。