Nucleocytoplasmic mRNA redistribution accompanies RNA binding protein mislocalization in ALS motor neurons and is restored by VCP ATPase inhibition

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Ziff et al. uncover widespread mRNA redistribution and mislocalization of RNA-binding proteins in ALS motor neurons. Their study highlights the potential of VCP ATPase inhibition in partially restoring mRNA and protein distribution, providing insights into ALS pathogenesis and a promising therapeutic target.