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Therapeutic Targeting of RNA Splicing in Cancer

Elizabeth A Bonner1,2, Stanley C Lee2,3

  • 1Molecular and Cellular Biology Graduate Program, University of Washington, Seattle, WA 98195, USA.

Genes
|July 29, 2023
PubMed

Insights

Aberrant RNA splicing, driven by spliceosome mutations, is implicated in hematologic malignancies. This review explores how altered splicing fuels cancer and discusses emerging RNA splicing-targeted therapies.

Area of Science:

  • Molecular Biology
  • Genetics
  • Oncology

Background:

  • RNA splicing is a critical gene expression regulator.
  • The spliceosome machinery controls RNA splicing.
  • Splicing dysregulation is linked to various diseases, including cancer.

Purpose of the Study:

  • To review the role of aberrant RNA splicing in cancer initiation and progression.
  • To discuss current and emerging therapeutic strategies targeting RNA splicing in cancer treatment.

Main Methods:

  • Literature review of RNA splicing mechanisms.
  • Analysis of spliceosome mutations in hematologic malignancies.
  • Examination of therapeutic approaches targeting RNA splicing.

Main Results:

  • Recurrent spliceosome mutations are identified in hematologic cancers.
  • Aberrant RNA splicing contributes to tumor development and progression.
  • Targeting RNA splicing presents a promising therapeutic avenue for cancer.

Conclusions:

  • Understanding splicing alterations is crucial for cancer pathogenesis.
  • Emerging technologies offer new ways to target RNA splicing for cancer therapy.
Keywords:
alternative splicinganti-sense oligonucleotidesimmuno-oncology therapiesspliceosome inhibitorssplicing factor mutations

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