2′-methoxy substituted oligomeric compounds and compositions for use in gene modulations
申请人:Ionis Pharmaceuticals, Inc.
公开号:US10478449B2
公开(公告)日:2019-11-19
Compositions comprising first and second oligomers are provided wherein at least a portion of the first oligomer is capable of hybridizing with at least a portion of the second oligomer, at least a portion of the first oligomer is complementary to and capable of hybridizing to a selected target nucleic acid, and at least one of the first or second oligomers includes a modified sugar and/or backbone modification. In some embodiments the modification is a 2′-OCH3 substituent group on a sugar moiety. Oligomer/protein compositions are also provided comprising an oligomer complementary to and capable of hybridizing to a selected target nucleic acid and at least one protein comprising at least a portion of an RNA-induced silencing complex (RISC), wherein at least one nucleotide of the oligomer has a modified sugar and/or backbone modification.
提供了包含第一和第二低聚物的组合物,其中第一低聚物的至少一部分能够与第二低聚物的至少一部分杂交,第一低聚物的至少一部分与选定的靶核酸互补并能够与靶核酸杂交,第一或第二低聚物中的至少一种包含修饰的糖和/或骨架修饰。在某些实施方案中,修饰是糖分子上的 2′-OCH3 取代基。还提供了寡聚体/蛋白质组合物,其中包含与所选靶核酸互补并能与之杂交的寡聚体和至少一种包含 RNA 诱导沉默复合体(RISC)至少一部分的蛋白质,其中寡聚体的至少一个核苷酸具有修饰的糖和/或骨架修饰。