Related Experiment Video

Updated: Jul 26, 2025

Engineering Artificial Factors to Specifically Manipulate Alternative Splicing in Human Cells
10:06

Engineering Artificial Factors to Specifically Manipulate Alternative Splicing in Human Cells

Published on: April 26, 2017

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Targeting pre-mRNA splicing in cancers: roles, inhibitors, and therapeutic opportunities

Shinsuke Araki1, Momoko Ohori1, Masato Yugami1

  • 1Research, Takeda Pharmaceutical Company Limited, Fujisawa, Kanagawa, Japan.

Frontiers in Oncology
|June 21, 2023
PubMed

Insights

Small-molecule splicing modulators offer a novel therapeutic approach for cancer by targeting pre-mRNA splicing. These modulators show promise in overcoming drug resistance and are advancing in clinical trials for various cancers.

Area of Science:

  • Molecular Biology
  • Oncology
  • Drug Discovery

Background:

  • Pre-mRNA splicing, particularly alternative splicing, is crucial in physiological processes and disease development.
  • Aberrant alternative splicing, driven by splicing factor mutations or altered expression, significantly contributes to cancer progression.
  • Cancer cells often develop resistance to conventional therapies, necessitating novel treatment strategies.

Purpose of the Study:

  • To review the current understanding of druggable splicing-related molecules in cancer.
  • To highlight the potential of small-molecule splicing modulators as a new class of cancer therapeutics.
  • To discuss future directions for splicing modulation in personalized and combination cancer therapies.

Main Methods:

  • Literature review of recent advancements in splicing modulation and cancer therapy.
  • Analysis of the role of splicing factors and their modulation in cancer progression.
  • Examination of clinical trial data for small-molecule splicing modulators.

Main Results:

  • Alternative splicing is a key driver in various cancers, offering therapeutic targets.
  • Small-molecule splicing modulators are emerging as promising anticancer agents, with several in clinical development.
  • Splicing modulation strategies can overcome resistance to conventional chemotherapy.

Conclusions:

  • Targeting pre-mRNA splicing represents a promising avenue for novel cancer therapies.
  • Small-molecule splicing modulators are advancing towards clinical application for diverse cancers.
  • Future cancer treatment paradigms should integrate personalized and combination strategies involving splicing modulation.
Keywords:
CDK12CLKSRPKalternative splicingpatient stratificationsmall moleculesplicing modulator

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