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Updated: May 30, 2025

Using the E1A Minigene Tool to Study mRNA Splicing Changes
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Targeting RNA splicing modulation: new perspectives for anticancer strategy?

Xuemei Lv1,2, Xiaoyu Sun1, Yang Gao1

  • 1Department of Pharmacology, School of Pharmacy, China Medical University, No.77 Puhe Road, Shenyang North New Area, Shenyang, Liaoning Province, 110122, P. R. China.

Journal of Experimental & Clinical Cancer Research : CR
|January 30, 2025
PubMed

Insights

Aberrant RNA splicing drives cancer development. This review explores cancer-specific splicing signatures and RNA splicing-targeted therapies, including splice-switching antisense oligonucleotides (ASOs), for improved cancer treatment.

Area of Science:

  • Molecular Biology
  • Genetics
  • Oncology

Background:

  • Alternative RNA splicing generates diverse proteins from a single gene.
  • Aberrant RNA splicing is a hallmark of most human cancers.
  • Targeting splicing offers novel therapeutic strategies for cancer treatment.

Purpose of the Study:

  • To review splice-altered signatures in cancer transcriptomes.
  • To discuss the role of splicing aberrations in tumorigenesis and progression.
  • To highlight RNA splicing-targeted cancer therapies and their clinical translation.

Main Methods:

  • Review of scientific literature on RNA splicing and cancer.
  • Analysis of cancer cell transcriptome data for splicing alterations.
  • Discussion of pharmacological agents and splice-switching antisense oligonucleotides (ASOs).

Main Results:

  • Identified distinct splice-altered signatures across various cancer types.
  • Detailed the contribution of splicing aberrations to cancer initiation and metastasis.
  • Summarized the mechanisms and clinical progress of splicing-targeted therapies.

Conclusions:

  • Aberrant RNA splicing is a critical driver of cancer.
  • RNA splicing-targeted strategies, including ASOs, show promise for cancer therapy.
  • Further research is needed to overcome challenges in clinical translation.
Keywords:
Alternative splicingCancerSplice-switching ASOSplicing modulatorsTargeted therapy

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