{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,26]],"date-time":"2026-03-26T07:17:54Z","timestamp":1774509474090,"version":"3.50.1"},"reference-count":19,"publisher":"Elsevier BV","issue":"3","license":[{"start":{"date-parts":[[2016,7,1]],"date-time":"2016-07-01T00:00:00Z","timestamp":1467331200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2016,7,1]],"date-time":"2016-07-01T00:00:00Z","timestamp":1467331200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"}],"funder":[{"DOI":"10.13039\/501100001691","name":"Japan Society for the Promotion of Science","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100001691","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001691","name":"Japan Society for the Promotion of Science","doi-asserted-by":"publisher","award":["26460221"],"award-info":[{"award-number":["26460221"]}],"id":[{"id":"10.13039\/501100001691","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001691","name":"Japan Society for the Promotion of Science","doi-asserted-by":"publisher","award":["23590642"],"award-info":[{"award-number":["23590642"]}],"id":[{"id":"10.13039\/501100001691","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Genetics and Metabolism"],"published-print":{"date-parts":[[2016,7]]},"DOI":"10.1016\/j.ymgme.2016.04.014","type":"journal-article","created":{"date-parts":[[2016,5,1]],"date-time":"2016-05-01T06:07:25Z","timestamp":1462082845000},"page":"214-219","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":20,"title":["In vitro evaluation of 2-hydroxyalkylated \u03b2-cyclodextrins as potential therapeutic agents for Niemann-Pick Type C disease"],"prefix":"10.1016","volume":"118","author":[{"given":"Yuki","family":"Kondo","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hiroko","family":"Tokumaru","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Yoichi","family":"Ishitsuka","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Tomoko","family":"Matsumoto","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Makiko","family":"Taguchi","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Keiichi","family":"Motoyama","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Taishi","family":"Higashi","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hidetoshi","family":"Arima","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Muneaki","family":"Matsuo","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Katsumi","family":"Higaki","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kousaku","family":"Ohno","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Tetsumi","family":"Irie","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"78","reference":[{"key":"10.1016\/j.ymgme.2016.04.014_bb0005","doi-asserted-by":"crossref","first-page":"16","DOI":"10.1186\/1750-1172-5-16","article-title":"Niemann-Pick disease type C","volume":"5","author":"Vanier","year":"2010","journal-title":"Orphanet J. Rare Dis."},{"key":"10.1016\/j.ymgme.2016.04.014_bb0010","doi-asserted-by":"crossref","first-page":"1721","DOI":"10.1126\/science.290.5497.1721","article-title":"How cells handle cholesterol","volume":"290","author":"Simons","year":"2000","journal-title":"Science"},{"key":"10.1016\/j.ymgme.2016.04.014_bb0015","doi-asserted-by":"crossref","first-page":"9404","DOI":"10.1523\/JNEUROSCI.1317-11.2011","article-title":"Unesterified cholesterol accumulation in late endosomes\/lysosomes causes neurodegeneration and is prevented by driving cholesterol export from this compartment","volume":"31","author":"Aqul","year":"2011","journal-title":"J. Neurosci."},{"key":"10.1016\/j.ymgme.2016.04.014_bb0020","series-title":"Cyclodext. Their Complexes","first-page":"1","article-title":"Molecules with holes \u2013 cyclodextrins","author":"Dodziuk","year":"2006"},{"key":"10.1016\/j.ymgme.2016.04.014_bb0025","doi-asserted-by":"crossref","first-page":"2377","DOI":"10.1073\/pnas.0810895106","article-title":"Reversal of defective lysosomal transport in NPC disease ameliorates liver dysfunction and neurodegeneration in the npc1\u2212\/\u2212 mouse","volume":"106","author":"Liu","year":"2009","journal-title":"Proc. Natl. Acad. Sci."},{"key":"10.1016\/j.ymgme.2016.04.014_bb0030","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0006951","article-title":"Chronic cyclodextrin treatment of murine Niemann-Pick C disease ameliorates neuronal cholesterol and glycosphingolipid storage and disease progression","volume":"4","author":"Davidson","year":"2009","journal-title":"PLoS ONE"},{"key":"10.1016\/j.ymgme.2016.04.014_bb0035","doi-asserted-by":"crossref","first-page":"309","DOI":"10.1203\/PDR.0b013e3181ee4dd2","article-title":"Weekly cyclodextrin administration normalizes cholesterol metabolism in nearly every organ of the Niemann-Pick type C1 mouse and markedly prolongs life","volume":"68","author":"Ramirez","year":"2010","journal-title":"Pediatr. Res."},{"key":"10.1016\/j.ymgme.2016.04.014_bb0040","doi-asserted-by":"crossref","first-page":"19316","DOI":"10.1073\/pnas.0910916106","article-title":"Cyclodextrin overcomes deficient lysosome-to-endoplasmic reticulum transport of cholesterol in Niemann-Pick type C cells","volume":"106","author":"Abi-Mosleh","year":"2009","journal-title":"Proc. Natl. Acad. Sci."},{"key":"10.1016\/j.ymgme.2016.04.014_bb0045","doi-asserted-by":"crossref","first-page":"76","DOI":"10.1016\/j.ymgme.2012.11.005","article-title":"Effects of cyclodextrin in two patients with Niemann\u2013Pick type C disease","volume":"108","author":"Matsuo","year":"2013","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymgme.2016.04.014_bb0050","first-page":"391","article-title":"Effects of intracerebroventricular administration of 2-hydroxypropyl-\u03b2-cyclodextrin in a patient with Niemann\u2013Pick type C disease","volume":"1","author":"Matsuo","year":"2014","journal-title":"Mol. Genet. Metab. Rep."},{"key":"10.1016\/j.ymgme.2016.04.014_bb0055","doi-asserted-by":"crossref","first-page":"844","DOI":"10.1248\/bpb.b14-00726","article-title":"Efficacy of 2-hydroxypropyl-\u03b2-cyclodextrin in Niemann\u2013Pick disease type C model mice and its pharmacokinetic analysis in a patient with the disease","volume":"38","author":"Tanaka","year":"2015","journal-title":"Biol. Pharm. Bull."},{"key":"10.1016\/j.ymgme.2016.04.014_bb0060","doi-asserted-by":"crossref","first-page":"875","DOI":"10.1093\/oxfordjournals.jbchem.a002932","article-title":"Isolation of NPC1-deficient Chinese hamster ovary cell mutants by gene trap mutagenesis","volume":"129","author":"Higaki","year":"2001","journal-title":"J. Biochem."},{"key":"10.1016\/j.ymgme.2016.04.014_bb0065","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0050262","article-title":"Sensitivity to lysosome-dependent cell death is directly regulated by lysosomal cholesterol content","volume":"7","author":"Appelqvist","year":"2012","journal-title":"PLoS ONE"},{"key":"10.1016\/j.ymgme.2016.04.014_bb0070","doi-asserted-by":"crossref","first-page":"78","DOI":"10.1023\/A:1016238720701","article-title":"Differential effects of sulfate and sulfobutyl ether of beta-cyclodextrin on erythrocyte membranes in vitro","volume":"12","author":"Shiotani","year":"1995","journal-title":"Pharm. Res."},{"key":"10.1016\/j.ymgme.2016.04.014_bb0075","first-page":"19","article-title":"Influence of Npc1 genotype on the toxicity of hydroxypropyl-\u03b2-cyclodextrin, a potentially therapeutic agent, in Niemann\u2013Pick type C disease models","volume":"1","author":"Tanaka","year":"2014","journal-title":"Mol. Genet. Metab. Rep."},{"key":"10.1016\/j.ymgme.2016.04.014_bb0080","doi-asserted-by":"crossref","first-page":"5477","DOI":"10.1073\/pnas.0914309107","article-title":"Endocytosis of beta-cyclodextrins is responsible for cholesterol reduction in Niemann-Pick type C mutant cells","volume":"107","author":"Rosenbaum","year":"2010","journal-title":"Proc. Natl. Acad. Sci."},{"key":"10.1016\/j.ymgme.2016.04.014_bb0085","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. Biol. Chem."},{"key":"10.1016\/j.ymgme.2016.04.014_bb0090","doi-asserted-by":"crossref","first-page":"2079","DOI":"10.3762\/bjoc.11.224","article-title":"Cholesterol lowering effects of mono-lactose-appended \u03b2-cyclodextrin in Niemann\u2013Pick type C disease-like HepG2 cells","volume":"11","author":"Motoyama","year":"2015","journal-title":"Beilstein J. Org. Chem."},{"key":"10.1016\/j.ymgme.2016.04.014_bb0095","doi-asserted-by":"crossref","DOI":"10.1038\/srep04356","article-title":"Lysosomal-specific cholesterol reduction by biocleavable polyrotaxanes for ameliorating Niemann-Pick type C disease","volume":"4","author":"Tamura","year":"2014","journal-title":"Sci. Rep."}],"updated-by":[{"DOI":"10.1016\/j.ymgme.2016.09.004","type":"erratum","label":"Erratum","source":"publisher","updated":{"date-parts":[[2017,3,1]],"date-time":"2017-03-01T00:00:00Z","timestamp":1488326400000}}],"container-title":["Molecular Genetics and Metabolism"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1096719216300579?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1096719216300579?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,10,21]],"date-time":"2025-10-21T23:21:09Z","timestamp":1761088869000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1096719216300579"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2016,7]]},"references-count":19,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2016,7]]}},"alternative-id":["S1096719216300579"],"URL":"https:\/\/doi.org\/10.1016\/j.ymgme.2016.04.014","relation":{"has-review":[{"id-type":"doi","id":"10.3410\/f.726367120.793542553","asserted-by":"object"}]},"ISSN":["1096-7192"],"issn-type":[{"value":"1096-7192","type":"print"}],"subject":[],"published":{"date-parts":[[2016,7]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"In vitro evaluation of 2-hydroxyalkylated \u03b2-cyclodextrins as potential therapeutic agents for Niemann-Pick Type C disease","name":"articletitle","label":"Article Title"},{"value":"Molecular Genetics and Metabolism","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.ymgme.2016.04.014","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2016 Elsevier Inc. All rights reserved.","name":"copyright","label":"Copyright"}]}}