Disney, Matthew D.,Liu, Biao,Childs-Disney, Jessica L.,Yang, Wang-Yong
申请号：
AU2013308578
公开号：
AU2013308578A1
申请日:
2013.08.30
申请国别(地区):
AU
年份:
2015
代理人:
摘要：
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 5C
