申请人:THE SCRIPPS RESEARCH INSTITUTE
公开号:US10961202B2
公开(公告)日:2021-03-30
Provided herein are compounds and methods for modulating abnormal repeat expansions of gene sequences. More particularly, provided are inhibitors of RNA and the uses of such inhibitors in regulating nucleotide repeat expansions, e.g., to treat Myotonic Dystrophy Type 1 (DM1), Myotonic Dystrophy Type 2 (DM2), Fuchs dystrophy, Huntington Disease, Amyotrophic Lateral Sclerosis, or Frontotemporal Dementia.
本文提供了用于调节基因序列异常重复扩增的化合物和方法。更具体地说,本文提供了 RNA 的抑制剂以及此类抑制剂在调节核苷酸重复扩增中的用途,例如,用于治疗肌营养不良症 1 型 (DM1)、肌营养不良症 2 型 (DM2)、福氏营养不良症、亨廷顿氏病、肌萎缩侧索硬化症或额叶痴呆症。