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Small Molecule Modulators of Pre-mRNA Splicing in Cancer Therapy

Maayan Salton1, Tom Misteli2

  • 1National Cancer Institute, National Institutes of Health, Bethesda, MD 20892, USA; Department of Biochemistry and Molecular Biology, the Institute for Medical Research Israel-Canada, Hebrew University-Hadassah Medical School, Jerusalem 91120, Israel.

Trends in Molecular Medicine
|December 25, 2015
PubMed

Insights

RNA splicing is crucial for gene expression and protein diversity, with defects linked to cancer. Small molecule splicing modulators offer a promising therapeutic strategy for cancer treatment by targeting specific splicing events.

Area of Science:

  • Molecular Biology
  • Cancer Research
  • Drug Discovery

Background:

  • Pre-messenger RNA (pre-mRNA) splicing is essential for mammalian gene expression.
  • Alternative RNA splicing significantly contributes to protein diversity.
  • Aberrant RNA splicing is implicated in numerous diseases, especially cancers, where tumors can be dependent on specific splicing isoforms.

Purpose of the Study:

  • To review the effects, mechanisms, and limitations of small molecule RNA splicing modulators.
  • To highlight the potential of targeting RNA processing as a therapeutic strategy in cancer treatment.
  • To discuss the development of selective splicing modulators to mitigate potential side effects.

Main Methods:

  • Literature review of reported RNA splicing modulators.
  • Analysis of their mechanisms of action.
  • Evaluation of their preclinical study outcomes and limitations.

Main Results:

  • Several RNA splicing modulators have been identified and show promise in preclinical studies.
  • While some modulators target general splicing machinery, leading to potential side effects, others demonstrate selectivity for specific splicing events.
  • Splicing defects are prevalent in cancer, underscoring the therapeutic potential of modulating RNA processing.

Conclusions:

  • Small molecule modulators of RNA processing represent a promising novel therapeutic strategy for cancer.
  • Targeting specific RNA splicing events offers a path toward more selective and effective cancer therapies.
  • Further research into the mechanisms and limitations of these modulators is crucial for clinical translation.
Keywords:
cancer therapysplicing modulators

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