Nature Communications (Mar 2022)

Indisulam targets RNA splicing and metabolism to serve as a therapeutic strategy for high-risk neuroblastoma

  • Anke Nijhuis,
  • Arti Sikka,
  • Orli Yogev,
  • Lili Herendi,
  • Cristina Balcells,
  • Yurui Ma,
  • Evon Poon,
  • Clare Eckold,
  • Gabriel N. Valbuena,
  • Yuewei Xu,
  • Yusong Liu,
  • Barbara Martins da Costa,
  • Michael Gruet,
  • Chiharu Wickremesinghe,
  • Adrian Benito,
  • Holger Kramer,
  • Alex Montoya,
  • David Carling,
  • Elizabeth J. Want,
  • Yann Jamin,
  • Louis Chesler,
  • Hector C. Keun

DOI
https://doi.org/10.1038/s41467-022-28907-3
Journal volume & issue
Vol. 13, no. 1
pp. 1 – 16

Abstract

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The prognosis of high-risk neuroblastoma is poor despite the availability of multimodal treatment. Here the authors show that high-risk neuroblastoma is sensitive to indisulam, a selective degrader of the splicing factor RBM39 through the dual targeting of RNA splicing and metabolism.