Enrichment of NPC1-deficient cells with the lipid LBPA stimulates autophagy, improves lysosomal function, and reduces cholesterol storage
Olga Ilnytska, Kimberly Lai, Kirill Gorshkov, Mark L. Schultz, Bruce Nguyen Tran, Maciej Jeziorek, Thaddeus J. Kunkel, Ruth D. Azaria, Hayley S. McLoughlin, Miriam Waghalter, Yang Xu, Michael Schlame, Nihal Altan‐Bonnet, Wei Zheng, Andrew P. Lieberman, Radek Dobrowolski, Judith Storch
Abstract
mouse model. Collectively, these findings provide a mechanistic basis supporting cellular LBPA as a potential new target for therapeutic intervention in NPC disease.
Topics & Concepts
NPC1BiologyAutophagyCholesterolMonoacylglycerol lipaseBiochemistryEndosomeLysosomeCell biologyEnzymeEndocannabinoid systemReceptorApoptosisLysosomal Storage Disorders ResearchCalcium signaling and nucleotide metabolismAutophagy in Disease and Therapy