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

Scott J Barraza,Anuradha Bhattacharyya,Christopher R Trotta,Matthew G Woll

Drug Discovery Today（2023）

Cited 2|Views4
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Abstract
•Modulation of pre-mRNA splicing is an effective alternative to the direct targeting of proteins.•Insight into pre-mRNA splicing biology offers practical targeted strategies to modulate splicing.•Since 2020, a surge of splicing-modulating small molecules have been discovered or developed.
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Key words
small molecule,alternative splicing,pre-mRNA,RNA,drug discovery,risdiplam,Evrysdi,kinase,regulatory element,epigenetic
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