[EN] MORPHOLINO MODIFIED OLIGOMERIC COMPOUNDS<br/>[FR] COMPOSÉS OLIGOMÈRES MODIFIÉS PAR MORPHOLINO
申请人:IONIS PHARMACEUTICALS INC
公开号:WO2018165564A1
公开(公告)日:2018-09-13
The present invention provides morpholino modified oligomeric compounds having at least one monomer subunit having Formula III, compounds having Formula I useful for making certain of the morpholino modified oligomeric compounds and methods of using the oligomeric compounds. In certain embodiments, the oligomeric compounds provided herein provide for an improved toxicity profile. Certain such oligomeric compounds are useful for hybridizing to a complementary nucleic acid, including but not limited, to nucleic acids in a cell. In certain embodiments, hybridization results in modulation of the amount of activity or expression of the target nucleic acid in a cell.
本发明提供了具有至少一个具有第III式的单体亚单位的形磷酸酯修饰寡聚合物化合物,具有用于制备某些形磷酸酯修饰寡聚合物化合物的第I式化合物以及使用寡聚合物化合物的方法。在某些实施例中,本文提供的寡聚合物化合物具有改进的毒性概况。某些这样的寡聚合物化合物适用于与细胞中的互补核酸杂交,包括但不限于核酸。在某些实施例中,杂交导致细胞中目标核酸的活性或表达量的调节。