Nature Communications (Oct 2017)

HDAC6 inhibition reverses axonal transport defects in motor neurons derived from FUS-ALS patients

  • Wenting Guo,
  • Maximilian Naujock,
  • Laura Fumagalli,
  • Tijs Vandoorne,
  • Pieter Baatsen,
  • Ruben Boon,
  • Laura Ordovás,
  • Abdulsamie Patel,
  • Marc Welters,
  • Thomas Vanwelden,
  • Natasja Geens,
  • Tine Tricot,
  • Veronick Benoy,
  • Jolien Steyaert,
  • Cynthia Lefebvre-Omar,
  • Werend Boesmans,
  • Matthew Jarpe,
  • Jared Sterneckert,
  • Florian Wegner,
  • Susanne Petri,
  • Delphine Bohl,
  • Pieter Vanden Berghe,
  • Wim Robberecht,
  • Philip Van Damme,
  • Catherine Verfaillie,
  • Ludo Van Den Bosch

DOI
https://doi.org/10.1038/s41467-017-00911-y
Journal volume & issue
Vol. 8, no. 1
pp. 1 – 15

Abstract

Read online

Amyotrophic lateral sclerosis (ALS) leads to selective loss of motor neurons. Using motor neurons derived from induced pluripotent stem cells from patients with ALS and FUS mutations, the authors demonstrate that axonal transport deficits that are observed in these cells can be rescued by HDAC6 inhibition.