Disclosed are RNA targeting compounds having the formula : wherein j is an integer from 1 to 100; each i is the same or different and is zero or an integer from 1 to 100; each Z1 represents the same or different linking moiety; each R1 is the same or different and represents an alkyl group or an aryl group; each Q1 represents the same or different RNA binding ligand; Q2 is an alkyl group; Q3 is a halogen, an alkyl group, an aryl group, or an amine. Also disclosed are RNA targeting compounds that include a polymer backbone and two or more pendant RNA binding ligands that are bound to the polymer backbone. Methods for using the subject RNA targeting compounds to treat myotonic dystrophy and other diseases are also disclosed, as are compounds that can be used to prepare the subject RNA targeting compounds.
公开了具有以下式子的 RNA 靶向化合物: 其中 j 是 1 至 100 的整数;每个 i 相同或不同,并且是零或 1 至 100 的整数;每个 Z1 代表相同或不同的连接基团;每个 R1 相同或不同,并且代表烷基或芳基;每个 Q1 代表相同或不同的 RNA 结合
配体;Q2 是烷基;Q3 是卤素、烷基、芳基或胺。还公开了包括聚合物骨架和与聚合物骨架结合的两种或两种以上垂体 RNA 结合
配体的 RNA 靶向化合物。还公开了使用主题 RNA 靶向化合物治疗肌营养不良症和其他疾病的方法,以及可用于制备主题 RNA 靶向化合物的化合物。