Neuronal network dysfunction precedes storage and neurodegeneration in a lysosomal storage disorder

Accumulation of lysosomal storage material and late-stage neurodegeneration are hallmarks of lysosomal storage disorders (LSDs) affecting the brain. Yet, for most LSDs, including CLN3 disease, the most common form of childhood dementia, it is unclear what mechanisms drive neurologic symptoms. Do def...

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Udgivet i:JCI Insight
Main Authors: Ahrens-Nicklas, Rebecca C., Tecedor, Luis, Hall, Arron F., Lysenko, Elena, Cohen, Akiva S., Davidson, Beverly L., Marsh, Eric D.
Format: Artigo
Sprog:Inglês
Udgivet: American Society for Clinical Investigation 2019
Fag:
Research Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6948765/
https://ncbi.nlm.nih.gov/pubmed/31573978
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/jci.insight.131961
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