Glucosylsphingosine is a potential fluid-based biomarker of lysosomal dysfunction in Cln3Δex7/8 mice

Glucosylsphingosine is a potential fluid-based biomarker of lysosomal dysfunction in Cln3Δex7/8 mice

CLN3 disease is a rare fatal juvenile neurodegenerative lysosomal storage disease. Challenges in tracking underlying disease biology have hindered the identification of effective therapeutic targets and the ability to execute clinical trials in this rare disease. While diagnostic biomarkers are read...

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Bibliographic Details
Main Authors: Hallie Wald, Stephanie Cicalese, Lihang Yao, Nathan Hatcher, Weiwei Luo, Xiaolan Shen, Xiaoli Ping, Brie Culp, Dan Metzger, Mark Ault, Christian Nunes, Mali Cosden, Sarah Jinn, Jason Uslaner, Sean Smith, Jacob Marcus, Robert Drolet
Format: Article
Language:English
Published: Elsevier 2025-10-01
Series:Neurobiology of Disease
Subjects:
Lysosome
Sphingolipid
Biomarker
CLN3 Batten disease
iPSC
Retina
Online Access:http://www.sciencedirect.com/science/article/pii/S0969996125002426
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http://www.sciencedirect.com/science/article/pii/S0969996125002426

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