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Compounds and methods for enhanced cellular uptake
专利权人:
Regulus Therapeutics Inc.
发明人:
Bhat Balkrishen
申请号:
NZ63092114
公开号:
NZ630921A
申请日:
2014.04.30
申请国别(地区):
NZ
年份:
2017
代理人:
摘要:
Disclosed herein are conjugated modified oligonucleotides of formula (I) that are complementary to a target RNA, wherein each N of Nm is, independently, a modified or unmodified nucleoside and m is 1 or 2; X1 and X2 are each, independently, a phosphodiester linkage or a phosphorothioate linkage; and MO is a modified oligonucleotide consisting of 8 to 25 linked nucleosides and comprising at least one nucleoside with a cEt modified sugar moiety, wherein the nucleobase sequence of the modified oligonucleotide is complementary to a microRNA. The conjugate facilitates cellular uptake of the modified oligonucleotide, resulting improved potency.
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