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COMPOSES OLIGONUCLEOTIDIQUES MULTIMERES
专利权人:
RANA THERAPEUTICS; INC.
发明人:
UHLMANN, EUGEN,WEBER, MARKUS,SUBRAMANIAN, ROMESH,ROCKEL, THOMAS DINO,KRIEG, ARTHUR M.
申请号:
CA2848753
公开号:
CA2848753A1
申请日:
2012.09.14
申请国别(地区):
CA
年份:
2013
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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