发明授权
US07291461B2 Methods for identifying small molecules that modulate premature translation termination and nonsense mRNA decay
有权
用于鉴定调节过早翻译终止和无义mRNA衰变的小分子的方法
- 专利标题： Methods for identifying small molecules that modulate premature translation termination and nonsense mRNA decay
- 专利标题（中）： 用于鉴定调节过早翻译终止和无义mRNA衰变的小分子的方法
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申请号： US10519243申请日： 2003-06-23
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公开(公告)号： US07291461B2公开(公告)日： 2007-11-06
- 发明人: Ellen Welch , Jin Zhuo
- 申请人： Ellen Welch , Jin Zhuo
- 申请人地址： US NJ South Plainfield
- 专利权人： PTC Therapeutics, Inc.
- 当前专利权人： PTC Therapeutics, Inc.
- 当前专利权人地址： US NJ South Plainfield
- 代理机构： Jones Day
- 国际申请： PCT/US03/19760 WO 20030623
- 国际公布： WO04/001010 WO 20031231
- 主分类号： C12Q1/68
- IPC分类号： C12Q1/68 ; C07H21/02 ; C07H21/04
摘要：
The present invention relates to methods for identifying compounds that modulate premature translation termination and/or nonsense-mediated mRNA decay by screening and identifying compounds that modulate the post-transcriptional expression of any gene with a premature translation stop codon. The invention particularly relates to using any gene encoding a premature stop codon to identify compounds that modulate premature translation termination and/or nonsense-mediated mRNA decay. A compound that modulates premature translation termination and/or nonsense-mediated mRNA decay of a target gene is identified using standard methods known in the art to measure changes in translation or mRNA stability of the gene product or mRNA of the gene with the premature stop codon. The methods of the present invention provide a simple, sensitive assay for high-throughput screening of libraries of compounds to identify pharmaceutical leads.
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