Antisense oligonucleotide-mediated MDM4 exon 6 skipping impairs tumor growth

J Clin Invest. 2016 Jan;126(1):68-84. doi: 10.1172/JCI82534. Epub 2015 Nov 23.

Abstract

MDM4 is a promising target for cancer therapy, as it is undetectable in most normal adult tissues but often upregulated in cancer cells to dampen p53 tumor-suppressor function. The mechanisms that underlie MDM4 upregulation in cancer cells are largely unknown. Here, we have shown that this key oncogenic event mainly depends on a specific alternative splicing switch. We determined that while a nonsense-mediated, decay-targeted isoform of MDM4 (MDM4-S) is produced in normal adult tissues as a result of exon 6 skipping, enhanced exon 6 inclusion leads to expression of full-length MDM4 in a large number of human cancers. Although this alternative splicing event is likely regulated by multiple splicing factors, we identified the SRSF3 oncoprotein as a key enhancer of exon 6 inclusion. In multiple human melanoma cell lines and in melanoma patient-derived xenograft (PDX) mouse models, antisense oligonucleotide-mediated (ASO-mediated) skipping of exon 6 decreased MDM4 abundance, inhibited melanoma growth, and enhanced sensitivity to MAPK-targeting therapeutics. Additionally, ASO-based MDM4 targeting reduced diffuse large B cell lymphoma PDX growth. As full-length MDM4 is enhanced in multiple human tumors, our data indicate that this strategy is applicable to a wide range of tumor types. We conclude that enhanced MDM4 exon 6 inclusion is a common oncogenic event and has potential as a clinically compatible therapeutic target.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Cell Cycle Proteins
  • Cell Line, Tumor
  • Cell Proliferation
  • Exons*
  • Humans
  • Melanoma / pathology
  • Melanoma / therapy*
  • Mice
  • Nuclear Proteins / genetics*
  • Oligonucleotides, Antisense / pharmacology*
  • Proto-Oncogene Proteins / genetics*
  • RNA-Binding Proteins / physiology
  • Serine-Arginine Splicing Factors
  • Tumor Suppressor Protein p53 / physiology

Substances

  • Cell Cycle Proteins
  • MDM4 protein, human
  • Nuclear Proteins
  • Oligonucleotides, Antisense
  • Proto-Oncogene Proteins
  • RNA-Binding Proteins
  • SRSF3 protein, human
  • Tumor Suppressor Protein p53
  • Serine-Arginine Splicing Factors