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Inhibitors of polynucleotide repeat-associated RNA foci and uses thereof
专利权人:
发明人:
Pascal Chartrand,Emmanuelle Querido
申请号:
US15024169
公开号:
US10092574B2
申请日:
2013.09.25
申请国别(地区):
US
年份:
2018
代理人:
摘要:
Compounds which inhibit the formation and/or accumulation of RNA foci, such as those due to polynucleotide repeats (e.g., trinucleotide repeats) are described herein. Also described herein are uses of such compounds, such as for the inhibition of the formation and accumulation such RNA foci, as well as for the treatment of polynucleotide repeat disorders (e.g., trinucleotide repeat disorders), such as myotonic dystrophy (e.g., DM1). Such compounds include compounds of formula 1, 1a, 1b, 2, 2a and (3) described herein.
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