Communications Biology (Sep 2021)

ALS-related FUS mutations alter axon growth in motoneurons and affect HuD/ELAVL4 and FMRP activity

  • Maria Giovanna Garone,
  • Nicol Birsa,
  • Maria Rosito,
  • Federico Salaris,
  • Michela Mochi,
  • Valeria de Turris,
  • Remya R. Nair,
  • Thomas J. Cunningham,
  • Elizabeth M. C. Fisher,
  • Mariangela Morlando,
  • Pietro Fratta,
  • Alessandro Rosa

Journal volume & issue
Vol. 4, no. 1
pp. 1 – 17


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Maria Giovanna Garone et al. use iPSC and mouse models to evaluate a mechanistic link between aberrant axonal phenotypes in ALS and the alteration of a cross-regulatory circuitry involving three RNA binding proteins: FUS, HuD and FMRP. Their results suggest NRN1 as a potential therapeutic target for ALS and provide further insight into the pathogenesis of this critical disorder.