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Methods of delivering multiple targeting oligonucleotides to a cell using cleavable linkers
专利权人:
Inc.;Translate Bio MA
发明人:
Eugen Uhlmann,Markus Weber,Romesh Subramanian,Thomas Dino Rockel,Arthur M. Krieg
申请号:
US14698312
公开号:
US09732341B2
申请日:
2015.04.28
申请国别(地区):
US
年份:
2017
代理人:
摘要:
The disclosure provides multimeric oligonucleotide compounds, comprising two or more target-specific oligonucleotides (e.g., antisense oligonucleotides (ASOs)), each being resistant to cleavage, and linked together by a cleavable linker. In particular, two or more linked target-specific oligonucleotides, each to a different target, allows concomitant inhibition of multiple genes' expression levels, while exhibiting favorable pharmacokinetic and pharmacodynamic properties. Methods of making and uses of the described compounds are also provided.
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