申请人:VALORISATION-RECHERCHE, LIMITED PARTNERSHIP
公开号:US20160213679A1
公开(公告)日:2016-07-28
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.
本文描述了抑制RNA斑点形成和/或积累的化合物,例如由多核苷酸重复(例如三核苷酸重复)引起的斑点。本文还描述了这些化合物的用途,例如用于抑制RNA斑点的形成和积累,以及用于治疗多核苷酸重复障碍(例如三核苷酸重复障碍),如肌萎缩性侧索硬化症(例如DM1)。这些化合物包括本文中描述的1、1a、1b、2、2a和(3)式的化合物。