发明申请
US20120190728A1 COMPOSITIONS AND METHODS FOR MODULATION OF SMN2 SPLICING IN A SUBJECT
有权
SMN2在主体中的分离调控的组成和方法
- 专利标题： COMPOSITIONS AND METHODS FOR MODULATION OF SMN2 SPLICING IN A SUBJECT
- 专利标题（中）： SMN2在主体中的分离调控的组成和方法
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申请号： US13380021申请日： 2010-06-17
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公开(公告)号： US20120190728A1公开(公告)日： 2012-07-26
- 发明人: C. Frank Bennett , Gene Hung , Frank Rigo , Adrian R. Krainer , Yimin Hua , Marco A. Passini , Lamya Shihabuddin , Seng H. Cheng , Katherine W. Klinger
- 申请人： C. Frank Bennett , Gene Hung , Frank Rigo , Adrian R. Krainer , Yimin Hua , Marco A. Passini , Lamya Shihabuddin , Seng H. Cheng , Katherine W. Klinger
- 国际申请： PCT/US10/39077 WO 20100617
- 主分类号： A61K31/713
- IPC分类号： A61K31/713 ; A61P25/28
摘要：
Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a subject. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy.
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