Modulators of alternative splicing as novel therapeutics in cancer

Sebastian Oltean

Research output: Contribution to journalArticle (Academic Journal)peer-review

5 Citations (Scopus)
287 Downloads (Pure)


Alternative splicing (AS), the process of removing introns from pre-mRNA and re-arrangement of exons to give several types of mature transcripts, has been described more than 40 years ago. However, until recently, it has not been clear how extensive it is. Genome-wide studies have now conclusively shown that more than 90% of genes are alternatively spliced in humans. This makes AS one of the main drivers of proteomic diversity and, consequently, determinant of cellular function repertoire. Unsurprisingly, given its extent, numerous splice isoforms have been described to be associated with several diseases including cancer. Many of them have antagonistic functions, e.g., pro- and anti-angiogenic or pro- and anti-apoptotic. Additionally several splice factors have been recently described to have oncogene or tumour suppressors activities, like SF3B1 which is frequently mutated in myelodysplastic syndromes. Beside the implications for cancer pathogenesis, de-regulated AS is recognized as one of the novel areas of cell biology where therapeutic manipulations may be designed. This editorial discusses the possibilities of manipulation of AS for therapeutic benefit in cancer. Approaches involving the use of oligonucleotides as well as small molecule splicing modulators are presented as well as thoughts on how specificity might be accomplished in splicing therapeutics.

Original languageEnglish
Pages (from-to)92-95
Number of pages4
JournalWorld Journal of Clinical Oncology
Issue number5
Early online date10 Oct 2015
Publication statusPublished - Oct 2015


  • Novel cancer therapeutics
  • Splicing switching oligonucleotides
  • Alternative splicing
  • Small molecules
  • Splicing modulators


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