Targeting strategies for modulating pre-mRNA splicing with small molecules: Recent advances

Barraza, SJ; Bhattacharyya, A; Trotta, CR; Woll, MG

Barraza, SJ (通讯作者),PTC Therapeut Inc, 100 Corp Court, South Plainfield, NJ 07080 USA.

DRUG DISCOVERY TODAY, 2023; 28 (1):

Abstract

The concept of using small molecules to therapeutically modulate pre-mRNA splicing was validated with the US Food and Drug Administration (FDA) approv......

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