Title : Transcriptome of HPβCD-treated Niemann-Pick disease type C1 cells highlights GPNMB as a biomarker for therapeutics.

Pub. Date : 2021 Nov 30

PMID : 34296265






1 Functional Relationships(s)
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1 The rare, fatal neurodegenerative disorder Niemann-Pick disease type C1 (NPC1) arises from lysosomal accumulation of unesterified cholesterol and glycosphingolipids. Glycosphingolipids NPC intracellular cholesterol transporter 1 Homo sapiens