Described herein are small molecule splicing modulator compounds that modulate splicing of mRNA, such as pre-mRNA, encoded by genes, and methods of use of the small molecule splicing modulator compounds for modulating splicing and treating diseases and conditions.
本文描述了一种小分子剪接调节剂化合物,可以调节由
基因编码的mRNA(如pre-mRNA)的剪接,以及利用这些小分子剪接调节剂化合物来调节剪接和治疗疾病和病况的方法。