发明名称 |
Small molecules targeting repeat r(CGG) sequences |
摘要 |
<p>The invention provides a series of bioactive small molecules that target expanded r(CGG) repeats, termed r(CGG)exp, that causes Fragile X-associated Tremor Ataxia Syndrome (FXTAS). The compound was identified by using information on the chemotypes and RNA motifs that interact. Specifically, 9-hydroxy-5,11-dimethyl-2-(2-(piperidin-1-yl)ethyl)-6H-pyrido[4,3-b]carbazol-2-ium, binds the 5'C</p> |
申请公布号 |
AU2013308578(A1) |
申请公布日期 |
2015.04.02 |
申请号 |
AU20130308578 |
申请日期 |
2013.08.30 |
申请人 |
THE SCRIPPS RESEARCH INSTITUTE |
发明人 |
DISNEY, MATTHEW D.;LIU, BIAO;CHILDS-DISNEY, JESSICA L.;YANG, WANG-YONG |
分类号 |
C12P17/04;A61K31/44;C12P17/10 |
主分类号 |
C12P17/04 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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