- 专利标题： OLIGONUCLEOTIDE ANALOGUES HAVING MODIFIED INTERSUBUNIT LINKAGES AND/OR TERMINAL GROUPS
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申请号： US16936906申请日： 2020-07-23
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公开(公告)号： US20210139897A1公开(公告)日： 2021-05-13
- 发明人: Gunnar J. Hanson , Alexander Charles Rudolph , Bao Zhong Cai , Ming Zhou , Dwight D. Weller
- 申请人： Sarepta Therapeutics, Inc.
- 申请人地址： US MA Cambridge
- 专利权人： Sarepta Therapeutics, Inc.
- 当前专利权人： Sarepta Therapeutics, Inc.
- 当前专利权人地址： US MA Cambridge
- 主分类号： C12N15/113
- IPC分类号： C12N15/113 ; C07F9/59 ; C07F9/572 ; C07F9/6558 ; C07F9/6561 ; C07F9/6584 ; C07H21/00 ; A61K31/496 ; A61K31/5377 ; C07D265/30 ; C07D295/108 ; C07F9/22 ; C07F9/26 ; C07F9/6544
摘要：
Oligonucleotide analogues comprising modified intersubunit linkages and/or modified 3′ and/or 5′-end groups are provided. The disclosed compounds are useful for the treatment of diseases where inhibition of protein expression or correction of aberrant mRNA splice products produces beneficial therapeutic effects.
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