Transcriptome-based screening in TARDBP/TDP-43 knock-in motor neurons identifies the NEDD8-activating enzyme inhibitor MLN4924

Transcriptome-based screening in TARDBP/TDP-43 knock-in motor neurons identifies the NEDD8-activating enzyme inhibitor MLN4924

Abstract A growing body of knowledge implicates perturbed RNA homeostasis in amyotrophic lateral sclerosis (ALS), a neurodegenerative disease that currently has no cure and few available treatments. Dysregulation of the multifunctional RNA-binding protein TDP-43 is increasingly regarded as a converg...

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Main Authors: Sarah Lépine, Gilles Maussion, Alexandria Schneider, Angela Nauleau-Javaudin, María José Castellanos-Montiel, Georgina Jiménez Ambriz, Dan Spiegelman, Narges Abdian, Anna Krystina Franco-Flores, Ghazal Haghi, Lale Gursu, Michael R. Fiorini, Allison A. Dilliott, Sali M. K. Farhan, Mathilde Chaineau, Thomas M. Durcan
Format: Article
Language:English
Published: Nature Portfolio 2025-08-01
Series:Scientific Reports
Online Access:https://doi.org/10.1038/s41598-025-12147-8
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https://doi.org/10.1038/s41598-025-12147-8

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