Gene therapy ameliorates neuromuscular pathology in CLN3 disease

Gene therapy ameliorates neuromuscular pathology in CLN3 disease

Abstract CLN3 disease is a neuronopathic lysosomal storage disorder that severely impacts the central nervous system (CNS) while also inducing notable peripheral neuromuscular symptoms. Although considerable attention has been directed towards the neurodegenerative consequences within the CNS, the i...

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Main Authors: Ewa A. Ziółkowska, Albina Jablonka-Shariff, Letitia L. Williams, Matthew J. Jansen, Sophie H. Wang, Elizabeth M. Eultgen, Matthew D. Wood, Daniel A. Hunter, Jaiprakash Sharma, Marco Sardiello, Robyn Reese, Alan Pestronk, Mark S. Sands, Alison K. Snyder-Warwick, Jonathan D. Cooper
Format: Article
Language:English
Published: BMC 2025-07-01
Series:Acta Neuropathologica Communications
Subjects:
CLN3 disease
Neuromuscular junction
Peripheral nervous system
Muscle atrophy
Terminal Schwann cells
AAV9 gene therapy
Online Access:https://doi.org/10.1186/s40478-025-02059-z
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https://doi.org/10.1186/s40478-025-02059-z

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