申请人:The General Hospital Corporation
公开号:US10676475B2
公开(公告)日:2020-06-09
Provided herein are compounds useful for improving mRNA splicing in a cell. Exemplary compounds provided herein are useful for improving mRNA splicing in genes comprising at least one exon ending in the nucleotide sequence CAA. Methods for preparing the compounds and methods of treating diseases of the central nervous system are also provided.
本文提供的化合物有助于改善细胞中的 mRNA 剪接。本文提供的示例化合物可用于改善包含至少一个以核苷酸序列 CAA 结尾的外显子的基因中的 mRNA 剪接。还提供了制备这些化合物的方法和治疗中枢神经系统疾病的方法。