ArticleTraffic (Copenhagen, Denmark)2010
Improvement in lipid and protein trafficking in Niemann-Pick C1 cells by correction of a secondary enzyme defect.
Article in Traffic (Copenhagen, Denmark), 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 45 papers.
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Who cites it
45 citing papers in PubMed, 82 citations in OpenAlex.
- Live-Cell Imaging and Lipidomics of Low-Density Lipoprotein Containing Intrinsically Fluorescent Cholesteryl Esters.Analytical chemistry · 2026Article
- Clinical and molecular spectrum of Niemann-Pick disease type C patients in India.Frontiers in pediatrics · 2026Article
- Lysosome dysfunction alters intestinal morphology and lipid metabolism in early neonatal piglets.BMC veterinary research · 2025Article
- Understanding the phenotypic variability in Niemann-Pick disease type C (NPC): a need for precision medicine.NPJ genomic medicine · 2023Review
- Kinetic modelling of sterol transport between plasma membrane and endo-lysosomes based on quantitative fluorescence and X-ray imaging data.Frontiers in cell and developmental biology · 2023Article
- Coronavirus Infection and Cholesterol Metabolism.Frontiers in immunology · 2022Review
- Review
- New paradigms for the treatment of lysosomal storage diseases: targeting the endocannabinoid system as a therapeutic strategy.Orphanet journal of rare diseases · 2021Review
- Inhibition of fatty acid amide hydrolase prevents pathology in neurovisceral acid sphingomyelinase deficiency by rescuing defective endocannabinoid signaling.EMBO molecular medicine · 2020Article
- Unbiased yeast screens identify cellular pathways affected in Niemann-Pick disease type C.Life science alliance · 2020Article
- The lysosome: A potential juncture between SARS-CoV-2 infectivity and Niemann-Pick disease type C, with therapeutic implications.FASEB journal : official publication of the Federation of American Societies for Experimental Biology · 2020Article
- Lipid-induced lysosomal damage after demyelination corrupts microglia protective function in lysosomal storage disorders.The EMBO journal · 2019Article
- Control of insulin granule formation and function by the ABC transporters ABCG1 and ABCA1 and by oxysterol binding protein OSBP.Molecular biology of the cell · 2018Article
- Altered Met receptor phosphorylation and LRP1-mediated uptake in cells lacking carbohydrate-dependent lysosomal targeting.The Journal of biological chemistry · 2017Article
- Lipids regulate the hydrolysis of membrane bound glucosylceramide by lysosomal β-glucocerebrosidase.Journal of lipid research · 2017Article
- Heat shock protein-based therapy as a potential candidate for treating the sphingolipidoses.Science translational medicine · 2016Article
- Complex lipid trafficking in Niemann-Pick disease type C.Journal of inherited metabolic disease · 2015Review
- Phosphatidylcholine: Greasing the Cholesterol Transport Machinery.Lipid insights · 2015Review
- Acid sphingomyelinase activity is regulated by membrane lipids and facilitates cholesterol transfer by NPC2.Journal of lipid research · 2014Article
- Lysosomal storage diseases and the heat shock response: convergences and therapeutic opportunities.Journal of lipid research · 2014Review
Corrections and comments
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Authors and funding
6 authors at 3 institutions in 1 country.
Funding
Abstract
Different primary lysosomal trafficking defects lead to common alterations in lipid trafficking, suggesting cooperative interactions among lysosomal lipids. However, cellular analysis of the functional consequences of this phenomenon is lacking. As a test case, we studied cells with defective Niemann-Pick C1 (NPC1) protein, a cholesterol trafficking protein whose defect gives rise to lysosomal accumulation of cholesterol and other lipids, leading to NPC disease. NPC1 cells also develop a secondary defect in acid sphingomyelinase (SMase) activity despite a normal acid SMase gene (SMPD1). When acid SMase activity was restored to normal levels in NPC1-deficient CHO cells through SMPD1 transfection, there was a dramatic reduction in lysosomal cholesterol. Two other defects, excess lysosomal bis-(monoacylglycerol) phosphate (BMP) and defective transferrin receptor (TfR) recycling, were also markedly improved. To test its relevance in human cells, the acid SMase activity defect in fibroblasts from NPC1 patients was corrected by SMPD1 transfection or acid SMase enzyme replacement. Both treatments resulted in a dramatic reduction in lysosomal cholesterol. These data show that correcting one aspect of a complex lysosomal lipid storage disease can reduce the cellular consequences even if the primary genetic defect is not corrected.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.