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Neuronal genetic rescue normalizes brain network dynamics in a lysosomal storage disorder despite persistent storage accumulation.
Mol Ther. 2022 Jul 6;30(7):2464-2473. doi: 10.1016/j.ymthe.2022.03.025. Epub 2022 Apr 5.
Mol Ther. 2022.
PMID: 35395398
Free PMC article.
Neuronal network dysfunction precedes storage and neurodegeneration in a lysosomal storage disorder.
Ahrens-Nicklas RC, Tecedor L, Hall AF, Lysenko E, Cohen AS, Davidson BL, Marsh ED.
Ahrens-Nicklas RC, et al.
JCI Insight. 2019 Nov 1;4(21):e131961. doi: 10.1172/jci.insight.131961.
JCI Insight. 2019.
PMID: 31573978
Free PMC article.
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CLN3 deficient cells display defects in the ARF1-Cdc42 pathway and actin-dependent events.
Schultz ML, Tecedor L, Stein CS, Stamnes MA, Davidson BL.
Schultz ML, et al.
PLoS One. 2014 May 2;9(5):e96647. doi: 10.1371/journal.pone.0096647. eCollection 2014.
PLoS One. 2014.
PMID: 24792215
Free PMC article.
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