Described herein are methods of use of small molecule splicing modulator compounds that modulate splicing of mRNA, such as pre-mRNA, encoded by genes, and methods of treating diseases and conditions associated with gene expression or activity of proteins encoded by genes.
本文描述了小分子剪接调节剂化合物的使用方法,这些化合物可调节
基因编码的 mRNA(如前 mRNA)的剪接,以及治疗与
基因表达或
基因编码的蛋白质活性相关的疾病和病症的方法。