Gene therapy ameliorates spontaneous seizures associated with cortical neuron loss in a Cln2(R207X) 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...

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Detalles Bibliográficos
Autores principales: Takahashi, Keigo, Eultgen, Elizabeth M., Wang, Sophie H., Rensing, Nicholas R., Nelvagal, Hemanth R., Dearborn, Joshua T., Danos, Olivier, Buss, Nicholas, Sands, Mark S., Wong, Michael, Cooper, Jonathan D.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society for Clinical Investigation 2023
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10266778/
https://www.ncbi.nlm.nih.gov/pubmed/37104037
http://dx.doi.org/10.1172/JCI165908

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