Nature Communications (Jul 2020)

Retromer stabilization results in neuroprotection in a model of Amyotrophic Lateral Sclerosis

  • Luca Muzio,
  • Riccardo Sirtori,
  • Davide Gornati,
  • Simona Eleuteri,
  • Andrea Fossaghi,
  • Diego Brancaccio,
  • Leonardo Manzoni,
  • Linda Ottoboni,
  • Luca De Feo,
  • Angelo Quattrini,
  • Eloise Mastrangelo,
  • Luca Sorrentino,
  • Emanuele Scalone,
  • Giancarlo Comi,
  • Luciana Marinelli,
  • Nilo Riva,
  • Mario Milani,
  • Pierfausto Seneci,
  • Gianvito Martino

DOI
https://doi.org/10.1038/s41467-020-17524-7
Journal volume & issue
Vol. 11, no. 1
pp. 1 – 17

Abstract

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ALS is a neurodegenerative disease characterized by loss of motor neurons. Here, the authors showed that reduced levels of the VSP35 subunit in the retromer complex is a conserved ALS feature and identified a new lead compound increasing retromer stability ameliorating the disease phenotype.