Neuronal genetic rescue normalizes brain network dynamics in a lysosomal storage disorder despite persistent storage accumulation
Author:
Funder
NICHD
NINDS
Publisher
Elsevier BV
Subject
Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine
Reference43 articles.
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3. A knock-in reporter model of Batten disease;Eliason;J. Neurosci.,2007
4. Hippocampal pathology in the human neuronal ceroid-lipofuscinoses: distinct patterns of storage deposition, neurodegeneration and glial activation;Tyynela;Brain Pathol.,2004
5. Combined anti-inflammatory and neuroprotective treatments have the potential to impact disease phenotypes in Cln3 (-/-) mice;Tarczyluk-Wells;Front. Neurol.,2019
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3. Gene therapy ameliorates spontaneous seizures associated with cortical neuron loss in a Cln2R207X mouse model;Journal of Clinical Investigation;2023-06-15
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