ETV6 dependency in Ewing sarcoma by antagonism of EWS-FLI1-mediated enhancer activation

Nat Cell Biol. 2023 Feb;25(2):298-308. doi: 10.1038/s41556-022-01060-1. Epub 2023 Jan 19.

Abstract

The EWS-FLI1 fusion oncoprotein deregulates transcription to initiate the paediatric cancer Ewing sarcoma. Here we used a domain-focused CRISPR screen to implicate the transcriptional repressor ETV6 as a unique dependency in this tumour. Using biochemical assays and epigenomics, we show that ETV6 competes with EWS-FLI1 for binding to select DNA elements enriched for short GGAA repeat sequences. Upon inactivating ETV6, EWS-FLI1 overtakes and hyper-activates these cis-elements to promote mesenchymal differentiation, with SOX11 being a key downstream target. We show that squelching of ETV6 with a dominant-interfering peptide phenocopies these effects and suppresses Ewing sarcoma growth in vivo. These findings reveal targeting of ETV6 as a strategy for neutralizing the EWS-FLI1 oncoprotein by reprogramming of genomic occupancy.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Cell Line, Tumor
  • Child
  • Gene Expression Regulation, Neoplastic
  • Humans
  • Oncogene Proteins, Fusion / genetics
  • Oncogene Proteins, Fusion / metabolism
  • Proto-Oncogene Protein c-fli-1 / genetics
  • Proto-Oncogene Protein c-fli-1 / metabolism
  • RNA-Binding Protein EWS / genetics
  • RNA-Binding Protein EWS / metabolism
  • Sarcoma, Ewing* / genetics
  • Sarcoma, Ewing* / metabolism
  • Sarcoma, Ewing* / pathology

Substances

  • EWS-FLI fusion protein
  • RNA-Binding Protein EWS
  • Proto-Oncogene Protein c-fli-1
  • Oncogene Proteins, Fusion