申请人:Berglund John Andrew
公开号:US20100323993A1
公开(公告)日:2010-12-23
Methods are provided herein for treatment of myotonic dystrophy and other toxic RNA diseases in a subject. In some examples, the method comprises administration of a compound that binds a nucleotide repeat expansion in a ribonucleic acid molecule, thereby treating the disease. In additional examples, the method comprises administration of a compound that disrupts binding of muscleblind-like proteins to an RNA nucleotide repeat expansion. Compounds for use in the disclosed method include pentamidine or heptamidine or derivatives thereof. Representative compounds are described herein.
本文提供了一种治疗肌无力萎缩症和其他毒性RNA疾病的方法。在某些例子中,该方法包括给受体注射一种化合物,该化合物能够结合核糖核酸分子中的核苷酸重复扩展,从而治疗该疾病。在其他例子中,该方法包括给受体注射一种化合物,该化合物能够破坏肌肉盲样蛋白与RNA核苷酸重复扩展的结合。用于该方法的化合物包括戊二酸二甲胺或庚二酸二甲胺或其衍生物。本文描述了代表性化合物。