{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,15]],"date-time":"2026-10-15T02:59:37Z","timestamp":1792033177964,"version":"4.4.0"},"reference-count":48,"publisher":"Elsevier BV","issue":"5","license":[{"start":{"date-parts":[[2013,12,1]],"date-time":"2013-12-01T00:00:00Z","timestamp":1385856000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2013,12,1]],"date-time":"2013-12-01T00:00:00Z","timestamp":1385856000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2013,10,28]],"date-time":"2013-10-28T00:00:00Z","timestamp":1382918400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/3.0\/"}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Cell Reports"],"published-print":{"date-parts":[[2013,12]]},"DOI":"10.1016\/j.celrep.2013.10.042","type":"journal-article","created":{"date-parts":[[2013,11,27]],"date-time":"2013-11-27T11:11:28Z","timestamp":1385550688000},"page":"1302-1315","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":292,"title":["Impaired Autophagy in the Lipid-Storage Disorder Niemann-Pick Type C1 Disease"],"prefix":"10.1016","volume":"5","author":[{"given":"Sovan","family":"Sarkar","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bernadette","family":"Carroll","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yosef","family":"Buganim","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dorothea","family":"Maetzel","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alex\u00a0H.M.","family":"Ng","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John P.","family":"Cassady","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Malkiel A.","family":"Cohen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Souvik","family":"Chakraborty","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Haoyi","family":"Wang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eric","family":"Spooner","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hidde","family":"Ploegh","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joerg","family":"Gsponer","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Viktor\u00a0I.","family":"Korolchuk","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rudolf","family":"Jaenisch","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.celrep.2013.10.042_bib1","doi-asserted-by":"crossref","first-page":"21883","DOI":"10.1074\/jbc.273.34.21883","article-title":"Isolation and characterization of rat liver amphisomes. Evidence for fusion of autophagosomes with both early and late endosomes","volume":"273","author":"Berg","year":"1998","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.celrep.2013.10.042_bib49","doi-asserted-by":"crossref","first-page":"2320","DOI":"10.1074\/jbc.270.5.2320","article-title":"Phosphorylation of ribosomal protein S6 is inhibitory for autophagy in isolated rat hepatocytes","volume":"270","author":"Blommaart","year":"1995","journal-title":"J. Biol. Chem."},{"key":"10.1016\/j.celrep.2013.10.042_bib2","doi-asserted-by":"crossref","first-page":"228","DOI":"10.1126\/science.277.5323.228","article-title":"Niemann-Pick C1 disease gene: homology to mediators of cholesterol homeostasis","volume":"277","author":"Carstea","year":"1997","journal-title":"Science"},{"key":"10.1016\/j.celrep.2013.10.042_bib3","doi-asserted-by":"crossref","first-page":"4876","DOI":"10.1093\/hmg\/dds324","article-title":"Impaired proteolysis underlies autophagic dysfunction in Niemann-Pick type C disease","volume":"21","author":"Elrick","year":"2012","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.celrep.2013.10.042_bib4","doi-asserted-by":"crossref","first-page":"1901","DOI":"10.1016\/j.bbamcr.2009.09.011","article-title":"TI-VAMP\/VAMP7 and VAMP3\/cellubrevin: two v-SNARE proteins involved in\u00a0specific steps of the autophagy\/multivesicular body pathways","volume":"1793","author":"Fader","year":"2009","journal-title":"Biochim. Biophys. Acta"},{"key":"10.1016\/j.celrep.2013.10.042_bib5","doi-asserted-by":"crossref","first-page":"885","DOI":"10.1038\/nature04724","article-title":"Suppression of basal autophagy in neural cells causes neurodegenerative disease in mice","volume":"441","author":"Hara","year":"2006","journal-title":"Nature"},{"key":"10.1016\/j.celrep.2013.10.042_bib6","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1006\/mgme.1999.2882","article-title":"Niemann-Pick C1 is a late endosome-resident protein that transiently associates with lysosomes and the trans-Golgi network","volume":"68","author":"Higgins","year":"1999","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.celrep.2013.10.042_bib7","doi-asserted-by":"crossref","first-page":"125","DOI":"10.1038\/nrm2336","article-title":"Cellular cholesterol trafficking and compartmentalization","volume":"9","author":"Ikonen","year":"2008","journal-title":"Nat. Rev. Mol. Cell Biol."},{"key":"10.1016\/j.celrep.2013.10.042_bib8","doi-asserted-by":"crossref","first-page":"954","DOI":"10.1016\/j.jocn.2008.09.020","article-title":"Association of autophagy with cholesterol-accumulated compartments in Niemann-Pick disease type C\u00a0cells","volume":"16","author":"Ishibashi","year":"2009","journal-title":"J.\u00a0Clin. Neurosci."},{"key":"10.1016\/j.celrep.2013.10.042_bib9","doi-asserted-by":"crossref","first-page":"1256","DOI":"10.1016\/j.cell.2012.11.001","article-title":"The hairpin-type tail-anchored SNARE syntaxin 17 targets to autophagosomes for fusion with endosomes\/lysosomes","volume":"151","author":"Itakura","year":"2012","journal-title":"Cell"},{"key":"10.1016\/j.celrep.2013.10.042_bib10","doi-asserted-by":"crossref","first-page":"4837","DOI":"10.1242\/jcs.01370","article-title":"Role for Rab7 in maturation of late autophagic vacuoles","volume":"117","author":"J\u00e4ger","year":"2004","journal-title":"J.\u00a0Cell Sci."},{"key":"10.1016\/j.celrep.2013.10.042_bib11","doi-asserted-by":"crossref","first-page":"5720","DOI":"10.1093\/emboj\/19.21.5720","article-title":"LC3, a mammalian homologue of yeast Apg8p, is localized in autophagosome membranes after processing","volume":"19","author":"Kabeya","year":"2000","journal-title":"EMBO J."},{"key":"10.1016\/j.celrep.2013.10.042_bib12","doi-asserted-by":"crossref","first-page":"659","DOI":"10.1016\/j.bbalip.2009.01.025","article-title":"Mechanisms and consequences of impaired lipid trafficking in Niemann-Pick type C1-deficient mammalian cells","volume":"1791","author":"Karten","year":"2009","journal-title":"Biochim. Biophys. Acta"},{"key":"10.1016\/j.celrep.2013.10.042_bib13","doi-asserted-by":"crossref","first-page":"452","DOI":"10.4161\/auto.4451","article-title":"Dissection of the autophagosome maturation process by a novel reporter protein, tandem fluorescent-tagged LC3","volume":"3","author":"Kimura","year":"2007","journal-title":"Autophagy"},{"key":"10.1016\/j.celrep.2013.10.042_bib14","doi-asserted-by":"crossref","first-page":"445","DOI":"10.4161\/auto.19496","article-title":"Guidelines for the use and interpretation of assays for monitoring autophagy","volume":"8","author":"Klionsky","year":"2012","journal-title":"Autophagy"},{"key":"10.1016\/j.celrep.2013.10.042_bib15","doi-asserted-by":"crossref","first-page":"113","DOI":"10.1038\/10084","article-title":"Late endosomal membranes rich in\u00a0lysobisphosphatidic acid regulate cholesterol transport","volume":"1","author":"Kobayashi","year":"1999","journal-title":"Nat. Cell Biol."},{"key":"10.1016\/j.celrep.2013.10.042_bib16","doi-asserted-by":"crossref","first-page":"3052","DOI":"10.1096\/fj.09-144519","article-title":"Altered lipid content inhibits autophagic vesicular fusion","volume":"24","author":"Koga","year":"2010","journal-title":"FASEB J."},{"key":"10.1016\/j.celrep.2013.10.042_bib17","doi-asserted-by":"crossref","first-page":"880","DOI":"10.1038\/nature04723","article-title":"Loss of autophagy in the central nervous system causes neurodegeneration in mice","volume":"441","author":"Komatsu","year":"2006","journal-title":"Nature"},{"key":"10.1016\/j.celrep.2013.10.042_bib18","doi-asserted-by":"crossref","first-page":"1149","DOI":"10.1016\/j.cell.2007.10.035","article-title":"Homeostatic levels of p62 control cytoplasmic inclusion body formation in autophagy-deficient mice","volume":"131","author":"Komatsu","year":"2007","journal-title":"Cell"},{"key":"10.1016\/j.celrep.2013.10.042_bib19","doi-asserted-by":"crossref","first-page":"213","DOI":"10.1038\/ncb2021","article-title":"The selective autophagy substrate p62 activates the stress responsive transcription factor Nrf2 through inactivation of Keap1","volume":"12","author":"Komatsu","year":"2010","journal-title":"Nat. Cell Biol."},{"key":"10.1016\/j.celrep.2013.10.042_bib20","doi-asserted-by":"crossref","first-page":"517","DOI":"10.1016\/j.molcel.2009.01.021","article-title":"Autophagy inhibition compromises degradation of ubiquitin-proteasome pathway substrates","volume":"33","author":"Korolchuk","year":"2009","journal-title":"Mol. Cell"},{"key":"10.1016\/j.celrep.2013.10.042_bib21","doi-asserted-by":"crossref","first-page":"1032","DOI":"10.1038\/nature03029","article-title":"The role of autophagy during the early neonatal starvation period","volume":"432","author":"Kuma","year":"2004","journal-title":"Nature"},{"key":"10.1016\/j.celrep.2013.10.042_bib22","doi-asserted-by":"crossref","first-page":"1213","DOI":"10.1016\/j.cell.2009.03.049","article-title":"Structure of N-terminal domain of NPC1 reveals distinct subdomains for binding and transfer of cholesterol","volume":"137","author":"Kwon","year":"2009","journal-title":"Cell"},{"key":"10.1016\/j.celrep.2013.10.042_bib24","doi-asserted-by":"crossref","first-page":"719","DOI":"10.4161\/auto.19469","article-title":"Autophagy in lysosomal storage disorders","volume":"8","author":"Lieberman","year":"2012","journal-title":"Autophagy"},{"key":"10.1016\/j.celrep.2013.10.042_bib25","doi-asserted-by":"crossref","first-page":"232","DOI":"10.1126\/science.277.5323.232","article-title":"Murine model of Niemann-Pick C disease: mutation in a cholesterol homeostasis gene","volume":"277","author":"Loftus","year":"1997","journal-title":"Science"},{"key":"10.1016\/j.celrep.2013.10.042_bib26","doi-asserted-by":"crossref","first-page":"411","DOI":"10.1016\/j.mam.2006.08.002","article-title":"Signalling and autophagy regulation in health, aging and disease","volume":"27","author":"Meijer","year":"2006","journal-title":"Mol. Aspects Med."},{"key":"10.1016\/j.celrep.2013.10.042_bib27","doi-asserted-by":"crossref","first-page":"38445","DOI":"10.1074\/jbc.M003180200","article-title":"Niemann-pick type C1 (NPC1) overexpression alters cellular cholesterol homeostasis","volume":"275","author":"Millard","year":"2000","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.celrep.2013.10.042_bib28","doi-asserted-by":"crossref","first-page":"1069","DOI":"10.1038\/nature06639","article-title":"Autophagy fights disease through cellular self-digestion","volume":"451","author":"Mizushima","year":"2008","journal-title":"Nature"},{"key":"10.1016\/j.celrep.2013.10.042_bib29","doi-asserted-by":"crossref","first-page":"2651","DOI":"10.1093\/hmg\/dds090","article-title":"Disruption and therapeutic rescue of autophagy in a human neuronal model of Niemann Pick type C1","volume":"21","author":"Ordonez","year":"2012","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.celrep.2013.10.042_bib30","doi-asserted-by":"crossref","first-page":"1495","DOI":"10.1093\/hmg\/ddm100","article-title":"Autophagy in Niemann-Pick C disease is dependent upon Beclin-1 and responsive to lipid trafficking defects","volume":"16","author":"Pacheco","year":"2007","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.celrep.2013.10.042_bib31","doi-asserted-by":"crossref","first-page":"24131","DOI":"10.1074\/jbc.M702824200","article-title":"p62\/SQSTM1 binds directly to\u00a0Atg8\/LC3 to facilitate degradation of ubiquitinated protein aggregates by autophagy","volume":"282","author":"Pankiv","year":"2007","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.celrep.2013.10.042_bib32","doi-asserted-by":"crossref","first-page":"9290","DOI":"10.1074\/jbc.M111.326405","article-title":"Normalization of cholesterol homeostasis by 2-hydroxypropyl-\u03b2-cyclodextrin in neurons and glia from Niemann-Pick C1 (NPC1)-deficient mice","volume":"287","author":"Peake","year":"2012","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.celrep.2013.10.042_bib33","doi-asserted-by":"crossref","first-page":"585","DOI":"10.1038\/ng1362","article-title":"Inhibition of mTOR induces autophagy and reduces toxicity of polyglutamine expansions in fly and mouse models of Huntington disease","volume":"36","author":"Ravikumar","year":"2004","journal-title":"Nat. Genet."},{"key":"10.1016\/j.celrep.2013.10.042_bib34","doi-asserted-by":"crossref","first-page":"1209","DOI":"10.1093\/hmg\/ddl036","article-title":"Rapamycin pre-treatment protects against apoptosis","volume":"15","author":"Ravikumar","year":"2006","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.celrep.2013.10.042_bib35","doi-asserted-by":"crossref","first-page":"1383","DOI":"10.1152\/physrev.00030.2009","article-title":"Regulation of mammalian autophagy in physiology and pathophysiology","volume":"90","author":"Ravikumar","year":"2010","journal-title":"Physiol. Rev."},{"key":"10.1016\/j.celrep.2013.10.042_bib36","doi-asserted-by":"crossref","first-page":"21","DOI":"10.2741\/2558","article-title":"Neural stem cell engineering: directed differentiation of adult and embryonic stem cells into neurons","volume":"13","author":"Robertson","year":"2008","journal-title":"Front. Biosci."},{"key":"10.1016\/j.celrep.2013.10.042_bib37","doi-asserted-by":"crossref","first-page":"789","DOI":"10.1111\/j.1471-4159.2010.06976.x","article-title":"Niemann-Pick type C disease:\u00a0molecular mechanisms and potential therapeutic approaches","volume":"116","author":"Rosenbaum","year":"2011","journal-title":"J.\u00a0Neurochem."},{"key":"10.1016\/j.celrep.2013.10.042_bib38","doi-asserted-by":"crossref","first-page":"709","DOI":"10.1038\/nrd3802","article-title":"Autophagy modulation as a potential therapeutic target for diverse diseases","volume":"11","author":"Rubinsztein","year":"2012","journal-title":"Nat. Rev. Drug Discov."},{"key":"10.1016\/j.celrep.2013.10.042_bib39","doi-asserted-by":"crossref","first-page":"1103","DOI":"10.1042\/BST20130134","article-title":"Regulation of autophagy by mTOR-dependent and mTOR-independent pathways: autophagy dysfunction in neurodegenerative diseases and therapeutic application of autophagy enhancers","volume":"41","author":"Sarkar","year":"2013","journal-title":"Biochem. Soc. Trans."},{"key":"10.1016\/j.celrep.2013.10.042_bib40","doi-asserted-by":"crossref","first-page":"1101","DOI":"10.1083\/jcb.200504035","article-title":"Lithium induces autophagy by inhibiting inositol monophosphatase","volume":"170","author":"Sarkar","year":"2005","journal-title":"J.\u00a0Cell Biol."},{"key":"10.1016\/j.celrep.2013.10.042_bib41","doi-asserted-by":"crossref","first-page":"331","DOI":"10.1038\/nchembio883","article-title":"Small molecules enhance autophagy and reduce toxicity in Huntington\u2019s disease models","volume":"3","author":"Sarkar","year":"2007","journal-title":"Nat. Chem. Biol."},{"key":"10.1016\/j.celrep.2013.10.042_bib42","doi-asserted-by":"crossref","first-page":"83","DOI":"10.1016\/S0076-6879(08)04005-6","article-title":"Autophagic clearance of aggregate-prone proteins associated with neurodegeneration","volume":"453","author":"Sarkar","year":"2009","journal-title":"Methods Enzymol."},{"key":"10.1016\/j.celrep.2013.10.042_bib43","doi-asserted-by":"crossref","first-page":"1131","DOI":"10.1038\/nature07976","article-title":"Autophagy regulates lipid metabolism","volume":"458","author":"Singh","year":"2009","journal-title":"Nature"},{"key":"10.1016\/j.celrep.2013.10.042_bib44","doi-asserted-by":"crossref","first-page":"3499","DOI":"10.1002\/pmic.201200205","article-title":"Proteomic analysis of mouse models of Niemann-Pick C disease reveals alterations in the steady-state levels of lysosomal proteins within the brain","volume":"12","author":"Sleat","year":"2012","journal-title":"Proteomics"},{"key":"10.1016\/j.celrep.2013.10.042_bib45","doi-asserted-by":"crossref","first-page":"513","DOI":"10.1038\/nrm2728","article-title":"Rab GTPases as coordinators of vesicle traffic","volume":"10","author":"Stenmark","year":"2009","journal-title":"Nat. Rev. Mol. Cell Biol."},{"key":"10.1016\/j.celrep.2013.10.042_bib46","doi-asserted-by":"crossref","first-page":"40","DOI":"10.1093\/hmg\/ddq430","article-title":"DJ-1 acts in parallel to the PINK1\/parkin pathway to control mitochondrial function and autophagy","volume":"20","author":"Thomas","year":"2011","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.celrep.2013.10.042_bib47","doi-asserted-by":"crossref","first-page":"9","DOI":"10.1038\/nrm3028","article-title":"Mechanisms of mitophagy","volume":"12","author":"Youle","year":"2011","journal-title":"Nat. Rev. Mol. Cell Biol."},{"key":"10.1016\/j.celrep.2013.10.042_bib48","doi-asserted-by":"crossref","first-page":"1371","DOI":"10.1016\/j.bpj.2009.06.025","article-title":"Roles of cholesterol in vesicle fusion and motion","volume":"97","author":"Zhang","year":"2009","journal-title":"Biophys. J."}],"container-title":["Cell Reports"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2211124713006414?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2211124713006414?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,10,31]],"date-time":"2025-10-31T09:27:13Z","timestamp":1761902833000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2211124713006414"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2013,12]]},"references-count":48,"journal-issue":{"issue":"5","published-print":{"date-parts":[[2013,12]]}},"alternative-id":["S2211124713006414"],"URL":"https:\/\/doi.org\/10.1016\/j.celrep.2013.10.042","relation":{},"ISSN":["2211-1247"],"issn-type":[{"value":"2211-1247","type":"print"}],"subject":[],"published":{"date-parts":[[2013,12]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Impaired Autophagy in the Lipid-Storage Disorder Niemann-Pick Type C1 Disease","name":"articletitle","label":"Article Title"},{"value":"Cell Reports","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.celrep.2013.10.042","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"Copyright \u00a9 2013 The Authors. Published by Elsevier Inc.","name":"copyright","label":"Copyright"}]}}