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Multimeric oligonucleotide compounds
专利权人:
Inc.;Translate Bio MA
发明人:
Uhlmann, Eugen,Subramanian, Romesh,Krieg, Arthur M.
申请号:
AU2013315225
公开号:
AU2013315225B2
申请日:
2013.09.13
申请国别(地区):
AU
年份:
2018
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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