Species-specific differences in NPC1 protein trafficking govern therapeutic response in Niemann-Pick type C disease

The folding and trafficking of transmembrane glycoproteins are essential for cellular homeostasis and are compromised in many diseases. In Niemann-Pick type C disease, a lysosomal disorder characterized by impaired intracellular cholesterol trafficking, the transmembrane glycoprotein NPC1 misfolds d...

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Bibliographic Details

Published in:	JCI Insight
Main Authors:	Mark L. Schultz, Kylie J. Schache, Ruth D. Azaria, Esmée Q. Kuiper, Steven Erwood, Evgueni A. Ivakine, Nicole Y. Farhat, Forbes D. Porter, Koralege C. Pathmasiri, Stephanie M. Cologna, Michael D. Uhler, Andrew P. Lieberman
Format:	Article
Language:	English
Published:	American Society for Clinical investigation 2022-12-01
Subjects:	Neuroscience Therapeutics
Online Access:	https://doi.org/10.1172/jci.insight.160308