Gene therapy ameliorates spontaneous seizures associated with cortical neuron loss in a Cln2R207X mouse model

Gene therapy ameliorates spontaneous seizures associated with cortical neuron loss in a Cln2R207X mouse model

Although a disease-modifying therapy for classic late infantile neuronal ceroid lipofuscinosis (CLN2 disease) exists, poor understanding of cellular pathophysiology has hampered the development of more effective and persistent therapies. Here, we investigated the nature and progression of neurologic...

Full description

Bibliographic Details
Main Authors: Keigo Takahashi, Elizabeth M. Eultgen, Sophie H. Wang, Nicholas R. Rensing, Hemanth R. Nelvagal, Joshua T. Dearborn, Olivier Danos, Nicholas Buss, Mark S. Sands, Michael Wong, Jonathan D. Cooper
Format: Article
Language:English
Published: American Society for Clinical Investigation 2023-06-01
Series:The Journal of Clinical Investigation
Subjects:
Neuroscience
Therapeutics
Online Access:https://doi.org/10.1172/JCI165908

Internet

https://doi.org/10.1172/JCI165908

Similar Items