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Updated: May 21, 2026

Engineering Artificial Factors to Specifically Manipulate Alternative Splicing in Human Cells
Published on: April 26, 2017
Splicing in oncogenesis and tumor suppression
Daisuke Kaida1, Tilman Schneider-Poetsch, Minoru Yoshida
1Frontier Research Core for Life Sciences, University of Toyama, Japan.
Abstract:
Post-transcriptional modifications, such as 5' end capping, 3' end polyadenylation and splicing, are necessary for the precise regulation of gene expression and transcriptome integrity. Therefore, it is not surprising that abnormalities of these post-transcriptional modifications prompt numerous diseases, including cancer. In fact, many studies revealed that misregulation of mRNA processing, especially splicing, are observed in a variety of cancer cells. In this review we describe how changes within RNA splicing regulatory elements or mutations in the processing factors alter the expression of tumor suppressors or oncogenes with pathological consequences. In addition, we show how several small molecules that bind to spliceosomal components and splicing regulators inhibit or modulate splicing activity. These compounds have anticancer activity and further development of small molecule modulators has potential in next generation cancer therapy.
Insights
Aberrant messenger RNA (mRNA) splicing is implicated in cancer development. Small molecules targeting splicing offer a promising avenue for novel cancer therapies.
Area of Science:
- Molecular Biology
- Cancer Biology
- RNA Biology
Background:
- Post-transcriptional modifications, including 5' capping, 3' polyadenylation, and splicing, are crucial for gene expression regulation and transcriptome stability.
- Dysregulation of these RNA processing events, particularly splicing, is frequently observed in cancer cells and contributes to disease pathogenesis.
Purpose of the Study:
- To review the mechanisms by which alterations in RNA splicing regulatory elements or mutations in splicing factors lead to altered expression of oncogenes and tumor suppressors.
- To explore the potential of small molecules that modulate splicing activity as a therapeutic strategy for cancer treatment.
Main Methods:
- Review of existing literature on RNA splicing, cancer, and small molecule therapeutics.
- Analysis of how changes in splicing impact oncogene and tumor suppressor expression.
- Examination of the anticancer activity of small molecules targeting spliceosomal components and splicing regulators.
Main Results:
- Alterations in RNA splicing regulatory elements or mutations in splicing factors can pathologically affect the expression of key cancer-related genes.
- Several small molecules have demonstrated the ability to inhibit or modulate splicing activity by interacting with spliceosomal components and splicing regulators.
Conclusions:
- Aberrant mRNA splicing is a significant factor in cancer development.
- Small molecule modulators of splicing exhibit anticancer activity and represent a promising area for next-generation cancer therapeutics.
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