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June 29, 2026Journal of Medicinal Chemistry

Recent Advances in Targeting RNA Splicing Factors with Small Molecules

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Authors

XYXinrui YuanJiangsu UniversityCYChao‐Yie YangUniversity of Tennessee Health Science Center

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Implication

Review examines small molecules targeting splicing factors to address aberrant splicing in cancers, implying potential therapeutic benefits.

Key Points

  • This review aims to highlight recent advances in targeting RNA splicing factors with small molecules for cancer treatment.
  • Reviewed mutations and dysregulated expressions of splicing factors in cancers.
  • Provided perspective on recent developments of small-molecule compounds.
  • Discussed functional assays facilitating hit/lead discovery for cancer treatment.
  • Identified key splicing factors (e.g., SF3B1, U2AF1) as drug targets.
  • Showed that targeting dysregulated splicing improves therapeutic outcomes in cancers.
  • Emphasized the role of small molecules in cancer treatment strategies.

Cite This Study

Yuan et al. (2026) studied this question.

synapsesocial.com/papers/6a420ab2f91bb43ea9191e6dhttps://doi.org/10.1021/acs.jmedchem.6c00234
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  1. 1Comprehensive molecular portraits of human breast tumours2012 · 12,630 citations
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  5. 5Autoregulation of RBM10 and cross-regulation of RBM10/RBM5 via alternative splicing-coupled nonsense-mediated decay2017 · 63 citations