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
Affiliations
- Wenting Guo
- KU Leuven-Department of Neurosciences, Experimental Neurology and Leuven Institute for Neuroscience and Disease (LIND)
- Maximilian Naujock
- Department of Neurology, Hannover Medical School
- Laura Fumagalli
- KU Leuven-Department of Neurosciences, Experimental Neurology and Leuven Institute for Neuroscience and Disease (LIND)
- Tijs Vandoorne
- KU Leuven-Department of Neurosciences, Experimental Neurology and Leuven Institute for Neuroscience and Disease (LIND)
- Pieter Baatsen
- VIB Bio Imaging Core and VIB-KU Leuven, Center for Brain and Disease Research
- Ruben Boon
- KU Leuven-Stem Cell Institute (SCIL)
- Laura Ordovás
- KU Leuven-Stem Cell Institute (SCIL)
- Abdulsamie Patel
- KU Leuven-Stem Cell Institute (SCIL)
- Marc Welters
- KU Leuven-Stem Cell Institute (SCIL)
- Thomas Vanwelden
- KU Leuven-Stem Cell Institute (SCIL)
- Natasja Geens
- KU Leuven-Department of Neurosciences, Experimental Neurology and Leuven Institute for Neuroscience and Disease (LIND)
- Tine Tricot
- KU Leuven-Stem Cell Institute (SCIL)
- Veronick Benoy
- KU Leuven-Department of Neurosciences, Experimental Neurology and Leuven Institute for Neuroscience and Disease (LIND)
- Jolien Steyaert
- KU Leuven-Department of Neurosciences, Experimental Neurology and Leuven Institute for Neuroscience and Disease (LIND)
- Cynthia Lefebvre-Omar
- Inserm U 1127, CNRS UMR 7225, Sorbonne Universités, UPMC Univ Paris 6, Institut du Cerveau et de la Moelle épinière (ICM), Hôpital Pitié-Salpêtrière
- Werend Boesmans
- Lab for Enteric NeuroScience, TARGID, KU Leuven
- Matthew Jarpe
- Acetylon Pharmaceuticals Inc.
- Jared Sterneckert
- Center for Regenerative Therapies Dresden (CRTD), Technische Universität Dresden
- Florian Wegner
- Department of Neurology, Hannover Medical School
- Susanne Petri
- Department of Neurology, Hannover Medical School
- Delphine Bohl
- Inserm U 1127, CNRS UMR 7225, Sorbonne Universités, UPMC Univ Paris 6, Institut du Cerveau et de la Moelle épinière (ICM), Hôpital Pitié-Salpêtrière
- Pieter Vanden Berghe
- Lab for Enteric NeuroScience, TARGID, KU Leuven
- Wim Robberecht
- KU Leuven-Department of Neurosciences, Experimental Neurology and Leuven Institute for Neuroscience and Disease (LIND)
- Philip Van Damme
- KU Leuven-Department of Neurosciences, Experimental Neurology and Leuven Institute for Neuroscience and Disease (LIND)
- Catherine Verfaillie
- KU Leuven-Stem Cell Institute (SCIL)
- Ludo Van Den Bosch
- KU Leuven-Department of Neurosciences, Experimental Neurology and Leuven Institute for Neuroscience and Disease (LIND)
- DOI
- https://doi.org/10.1038/s41467-017-00911-y
- Journal volume & issue
-
Vol. 8,
no. 1
pp. 1 – 15
Abstract
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.