Viral-mediated knockdown of Atxn2 attenuates TDP-43 pathology and muscle dysfunction in the PFN1C71G ALS mouse model

Viral-mediated knockdown of Atxn2 attenuates TDP-43 pathology and muscle dysfunction in the PFN1C71G ALS mouse model

Abstract Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder characterized by progressive motor neuron loss and muscle atrophy. Hyperphosphorylated aggregation of the RNA-binding protein, TDP-43, in the motor cortex and spinal cord are defining molecular features of ALS, sugges...

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Main Authors: Zachary C. E. Hawley, Xueying Li, Dora Bodnar, Yuanzheng Gu, Yi Luo, Daniel Ferretti, Adam Sheehy, Rachelle Driscoll, Maria I. Zavodszky, Shaolong Cao, Isabel Isaza, Luke Jandreski, Yuqing Liu, Thomas Carlile, Shih-Ching Lo, Anna Grimard, Shawn Bourque, Aditya Utturkar, Samantha Desmarais, H. Moore Arnold, Dann Huh, Edward Guilmette, Deborah Y. Kwon
Format: Article
Language:English
Published: BMC 2025-05-01
Series:Acta Neuropathologica Communications
Online Access:https://doi.org/10.1186/s40478-025-02005-z
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