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Pharmaceutical compositions and treatment of genetic diseases associated with nonsense mediated RNA decay
专利权人:
Timothy J. Cardozo;Lawrence B. Gardner;Leenus Martin
发明人:
Lawrence B. Gardner,Timothy J. Cardozo,Leenus Martin
申请号:
US14044457
公开号:
US09216180B2
申请日:
2013.10.02
申请国别(地区):
US
年份:
2015
代理人:
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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中国工程科技知识中心
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