公开(公告)号：US20210163934A1

公开(公告)日：2021-06-03

申请号：US16982758

申请日：2019-03-22

申请人： NATIONAL UNIVERSITY CORPORATION TOKYO MEDICAL AND DENTAL UNIVERSITY

发明人： Takanori YOKOTA ,  Tetsuya NAGATA ,  Hideki FURUKAWA ,  Yasuo NAKAGAWA ,  Takatoshi YOGO ,  Ryosuke TOKUNOH ,  Shigekazu SASAKI ,  Kosuke HIDAKA ,  Tomohiro SEKI ,  Kenichi MIYATA ,  Yusuke ADACHI ,  Naoto ARIMURA

IPC分类号： C12N15/113 ,  A61P25/28 ,  A61K31/713

摘要： The object of the present invention is to provide a nucleic acid agent that can be efficiently delivered to the nervous system, particularly the central nervous system to which the BBB mechanism prevents drug delivery, and can produce an antisense effect on a target transcriptional product at the delivered site, and a composition comprising the same.
Provided is a double-stranded nucleic acid complex formed by annealing a first nucleic acid strand capable of hybridizing to part of a target transcriptional product, and has an antisense effect on the target transcriptional product, to a second nucleic acid strand comprising a base sequence complementary to the first nucleic acid strand, and is bound to tocopherol or an analog thereof, cholesterol or an analog thereof, or a substituted or unsubstituted C1-30 alkyl group, a substituted or unsubstituted C2-30 alkenyl group, or a substituted or unsubstituted C1-30 alkoxy group.