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.
本文描述了可调节
基因编码的 mRNA(如 pre-mRNA)剪接的小分子剪接调节剂化合物,以及使用小分子剪接调节剂化合物调节剪接和治疗疾病的方法。