发明授权
US09216180B2 Pharmaceutical compositions and treatment of genetic diseases associated with nonsense mediated RNA decay
有权
与无义介导的RNA衰变相关的遗传疾病的药物组合物和治疗
- 专利标题： Pharmaceutical compositions and treatment of genetic diseases associated with nonsense mediated RNA decay
- 专利标题（中）： 与无义介导的RNA衰变相关的遗传疾病的药物组合物和治疗
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申请号： US14044457申请日： 2013-10-02
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公开(公告)号： US09216180B2公开(公告)日： 2015-12-22
- 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
- 代理机构： LeClairRyan, a Professional Corporation
- 主分类号： A61K31/535
- IPC分类号： A61K31/535 ; A61K31/415 ; A61K31/505 ; A61K31/53 ; A61K31/4152 ; A61K45/06 ; G01N33/50 ; G06F19/12 ; A61K31/167 ; A61K31/34 ; A61K31/381 ; A61K31/4035 ; A61K31/4184 ; A61K31/4192 ; A61K31/433 ; A61K31/445 ; A61K31/513 ; A61K31/538 ; G06F19/16 ; G06F19/00
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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