发明申请
US20140094457A1 PHARMACEUTICAL COMPOSITIONS AND TREATMENT OF GENETIC DISEASES ASSOCIATED WITH NONSENSE MEDIATED RNA DECAY
有权
药物组合物和与非神经介导的RNA衰减相关的遗传病的治疗
- 专利标题： PHARMACEUTICAL COMPOSITIONS AND TREATMENT OF GENETIC DISEASES ASSOCIATED WITH NONSENSE MEDIATED RNA DECAY
- 专利标题（中）： 药物组合物和与非神经介导的RNA衰减相关的遗传病的治疗
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申请号： US14044457申请日： 2013-10-02
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公开(公告)号： US20140094457A1公开(公告)日： 2014-04-03
- 发明人: Lawrence B. Gardner , Timothy J. Cardozo , Leenus Martin
- 申请人： Lawrence B. Gardner , Timothy J. Cardozo , Leenus Martin
- 申请人地址： US NY New York
- 专利权人： NEW YORK UNIVERSITY
- 当前专利权人： NEW YORK UNIVERSITY
- 当前专利权人地址： US NY New York
- 主分类号： A61K31/53
- IPC分类号： A61K31/53 ; G06F19/12 ; G01N33/50 ; A61K31/4152 ; A61K45/06
摘要：
The present invention relates to pharmaceutical compositions comprising a compound and a pharmaceutically acceptable carrier. The present invention is also directed to a method of treating a genetic disease caused by premature termination codons, or other conditions that render messenger ribonucleic acid (mRNA) susceptible to nonsense mediated RNA decay, in a subject. Also disclosed is a method of inhibiting nonsense mediated RNA decay and/or induction of autophagy. The present invention also relates to a method of identifying inhibitors of nonsense mediated RNA decay and/or inducing autophagy. The present invention further relates to a method of inhibiting nonsense mediated RNA decay and/or induction of autophagy in a subject.
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