RNA剪接
计算生物学
药物发现
小分子
生物
食品药品监督管理局
信使核糖核酸
核糖核酸
生物信息学
基因
遗传学
药理学
作者
Scott J. Barraza,Anuradha Bhattacharyya,Christopher Trotta,Matthew G. Woll
标识
DOI:10.1016/j.drudis.2022.103431
摘要
The concept of using small molecules to therapeutically modulate pre-mRNA splicing was validated with the US Food and Drug Administration (FDA) approval of Evrysdi® (risdiplam) in 2020. Since then, efforts have continued unabated toward the discovery of new splicing-modulating drugs. However, the drug development world has evolved in the 10 years since risdiplam precursors were first identified in high-throughput screening (HTS). Now, new mechanistic insights into RNA-processing pathways and regulatory networks afford increasingly feasible targeted approaches. In this review, organized into classes of biological target, we compile and summarize small molecules discovered, devised, and developed since 2020 to alter pre-mRNA splicing.
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