发明名称 Compounds and methods for enhanced cellular uptake
摘要 Described herein are conjugated modified oligonucleotides that are complementary to a target RNA. The conjugate facilitates cellular uptake of the modified oligonucleotide, resulting improved potency.
申请公布号 US9506030(B2) 申请公布日期 2016.11.29
申请号 US201414266127 申请日期 2014.04.30
申请人 Regulus Therapeutics Inc. 发明人 Bhat Balkrishen
分类号 C07H17/02;C07H21/02;C12N5/00;C12N5/02;C12N5/071;C12N15/11;C12N15/113 主分类号 C07H17/02
代理机构 McNeill Baur PLLC 代理人 McNeill Baur PLLC
主权项 1. A compound comprising the structure: wherein each N of Nm is, independently, a modified or unmodified nucleoside and m is from 1 to 5; X1 and X2 are each, independently, a phosphodiester linkage or a phosphorothioate linkage; and MO is a modified oligonucleotide; and wherein when m is greater than 1, each modified or unmodified nucleoside of Nm is connected to adjacent modified or unmodified nucleosides of Nm by a phosphodiester internucleoside linkage or a phosphorothioate internucleoside linkage.
地址 San Diego CA US