{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,2,25]],"date-time":"2026-02-25T09:33:29Z","timestamp":1772012009938,"version":"3.50.1"},"reference-count":48,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2013,11,1]],"date-time":"2013-11-01T00:00:00Z","timestamp":1383264000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Neuroscience"],"published-print":{"date-parts":[[2013,11]]},"DOI":"10.1016\/j.neuroscience.2013.08.001","type":"journal-article","created":{"date-parts":[[2013,8,13]],"date-time":"2013-08-13T04:00:28Z","timestamp":1376366428000},"page":"201-211","source":"Crossref","is-referenced-by-count":30,"special_numbering":"C","title":["Combined therapy with cyclodextrin\/allopregnanolone and miglustat improves motor but not cognitive functions in Niemann\u2013Pick Type C1 mice"],"prefix":"10.1016","volume":"252","author":[{"given":"M.","family":"Hovakimyan","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"F.","family":"Maass","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"J.","family":"Petersen","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"C.","family":"Holzmann","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"M.","family":"Witt","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"J.","family":"Lukas","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"M.J.","family":"Frech","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"R.","family":"H\u00fcbner","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"A.","family":"Rolfs","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"A.","family":"Wree","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"78","reference":[{"key":"10.1016\/j.neuroscience.2013.08.001_b0005","doi-asserted-by":"crossref","first-page":"811","DOI":"10.1002\/jnr.20685","article-title":"Allopregnanolone treatment, both as a single injection or repetitively, delays demyelination and enhances survival of Niemann\u2013Pick C mice","volume":"82","author":"Ahmad","year":"2005","journal-title":"J Neurosci Res"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0010","doi-asserted-by":"crossref","first-page":"269","DOI":"10.1007\/BF03195222","article-title":"Neural stem cell implantation extends life in Niemann\u2013Pick C1 mice","volume":"48","author":"Ahmad","year":"2007","journal-title":"J Appl Genet"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0015","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.neuroscience.2013.08.001_b0020","doi-asserted-by":"crossref","first-page":"38","DOI":"10.1016\/S0006-8993(99)01586-3","article-title":"A behavioral and neuroanatomical assessment of an inbred substrain of 129 mice with behavioral comparisons to C57BL\/6J mice","volume":"836","author":"Balogh","year":"1999","journal-title":"Brain Res"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0025","doi-asserted-by":"crossref","first-page":"887","DOI":"10.1016\/S0014-4886(03)00345-5","article-title":"Postnatal development of inflammation in a murine model of Niemann\u2013Pick type C disease: immunohistochemical observations of microglia and astroglia","volume":"184","author":"Baudry","year":"2003","journal-title":"Exp Neurol"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0030","doi-asserted-by":"crossref","first-page":"39","DOI":"10.1016\/j.jchemneu.2011.04.003","article-title":"Alteration of the CNS pathway to the hippocampus in a mouse model of Niemann\u2013Pick, type C disease","volume":"42","author":"Byun","year":"2011","journal-title":"J Chem Neuroanat"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0035","doi-asserted-by":"crossref","first-page":"131","DOI":"10.1016\/S0024-3205(01)01384-4","article-title":"Cyclodextrins in the treatment of a mouse model of Niemann\u2013Pick C disease","volume":"70","author":"Camargo","year":"2001","journal-title":"Life Sci"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0040","doi-asserted-by":"crossref","first-page":"228","DOI":"10.1126\/science.277.5323.228","article-title":"Niemann\u2013Pick C1 disease gene: homology to mediators of cholesterol homeostasis","volume":"277","author":"Carstea","year":"1997","journal-title":"Science"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0045","doi-asserted-by":"crossref","first-page":"1509","DOI":"10.1002\/glia.20563","article-title":"Decreased estradiol release from astrocytes contributes to the neurodegeneration in a mouse model of Niemann\u2013Pick disease type C","volume":"55","author":"Chen","year":"2007","journal-title":"Glia"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0050","doi-asserted-by":"crossref","first-page":"1541","DOI":"10.1172\/JCI0215420","article-title":"Rab proteins mediate Golgi transport of caveola-internalized glycosphingolipids and correct lipid trafficking in Niemann\u2013Pick C cells","volume":"109","author":"Choudhury","year":"2002","journal-title":"J Clin Invest"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0055","doi-asserted-by":"crossref","first-page":"e6951","DOI":"10.1371\/journal.pone.0006951","article-title":"Chronic cyclodextrin treatment of murine Niemann\u2013Pick 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.neuroscience.2013.08.001_b0060","doi-asserted-by":"crossref","first-page":"e23677","DOI":"10.1371\/journal.pone.0023677","article-title":"Loss of Niemann\u2013Pick C1 or C2 protein results in similar biochemical changes suggesting that these proteins function in a common lysosomal pathway","volume":"6","author":"Dixit","year":"2011","journal-title":"PLoS One"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0065","doi-asserted-by":"crossref","first-page":"6394","DOI":"10.1523\/JNEUROSCI.4909-08.2009","article-title":"Mitochondrial cholesterol loading exacerbates amyloid beta peptide-induced inflammation and neurotoxicity","volume":"29","author":"Fern\u00e1ndez","year":"2009","journal-title":"J Neurosci"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0070","doi-asserted-by":"crossref","first-page":"214","DOI":"10.1016\/j.ymgme.2010.06.018","article-title":"Oxidative stress in Niemann\u2013Pick disease, type C","volume":"101","author":"Fu","year":"2010","journal-title":"Mol Genet Metab"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0075","doi-asserted-by":"crossref","first-page":"415","DOI":"10.1002\/cne.1149","article-title":"Selective neurodegeneration, without neurofibrillary tangles, in a mouse model of Niemann\u2013Pick C disease","volume":"433","author":"German","year":"2001","journal-title":"J Comp Neurol"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0080","doi-asserted-by":"crossref","first-page":"704","DOI":"10.1038\/nm1073","article-title":"Niemann\u2013Pick type C disease involves disrupted neurosteroidogenesis and responds to allopregnanolone","volume":"10","author":"Griffin","year":"2004","journal-title":"Nat Med"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0085","doi-asserted-by":"crossref","first-page":"175","DOI":"10.1007\/BF00227765","article-title":"Cerebellar degeneration in the Niemann\u2013Pick type C mouse","volume":"85","author":"Higashi","year":"1993","journal-title":"Acta Neuropathol"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0090","doi-asserted-by":"crossref","first-page":"e28418","DOI":"10.1371\/journal.pone.0028418","article-title":"Corneal alterations during combined therapy with cyclodextrin\/allopregnanolone and miglustat in a knock-out mouse model of NPC1 disease","volume":"6","author":"Hovakimyan","year":"2011","journal-title":"PLoS One"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0095","doi-asserted-by":"crossref","first-page":"69","DOI":"10.1078\/0940-2993-00301","article-title":"Behavioral phenotyping of mice in pharmacological and toxicological research","volume":"55","author":"Karl","year":"2003","journal-title":"Exp Toxicol Pathol"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0100","doi-asserted-by":"crossref","first-page":"455","DOI":"10.1111\/j.1749-6632.2002.tb07587.x","article-title":"Modeling cerebellar flocculus and paraflocculus involvement in complex predictive smooth eye pursuit in monkeys","volume":"978","author":"Kettner","year":"2002","journal-title":"Ann N Y Acad Sci"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0105","doi-asserted-by":"crossref","first-page":"50","DOI":"10.1055\/s-2008-1034748","article-title":"Eye movement abnormalities as a sign for the diagnosis in Niemann\u2013Pick disease type C","volume":"214","author":"Lengyel","year":"1999","journal-title":"Klin Monbl Augenheilkd"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0110","doi-asserted-by":"crossref","first-page":"323","DOI":"10.1093\/jnen\/64.4.323","article-title":"Molecular, anatomical, and biochemical events associated with neurodegeneration in mice with Niemann\u2013Pick type C disease","volume":"64","author":"Li","year":"2005","journal-title":"J Neuropathol Exp Neurol"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0115","doi-asserted-by":"crossref","first-page":"962","DOI":"10.2353\/ajpath.2007.070052","article-title":"Cholesterol accumulation is associated with lysosomal dysfunction and autophagic stress in Npc1\u2212\/\u2212 mouse brain","volume":"171","author":"Liao","year":"2007","journal-title":"Am J Pathol"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0120","doi-asserted-by":"crossref","first-page":"933","DOI":"10.1194\/jlr.M000257","article-title":"Cyclodextrin overcomes the transport defect in nearly every organ of NPC1 mice leading to excretion of sequestered cholesterol as bile acid","volume":"51","author":"Liu","year":"2010","journal-title":"J Lipid Res"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0125","doi-asserted-by":"crossref","first-page":"232","DOI":"10.1126\/science.277.5323.232","article-title":"Murine model of Niemann\u2013Pick C disease: mutation in a cholesterol homeostasis gene","volume":"277","author":"Loftus","year":"1997","journal-title":"Science"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0130","doi-asserted-by":"crossref","first-page":"891","DOI":"10.1172\/JCI0216500","article-title":"Intracellular cholesterol transport","volume":"110","author":"Maxfield","year":"2002","journal-title":"J Clin Invest"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0135","doi-asserted-by":"crossref","first-page":"410","DOI":"10.1016\/j.brainresrev.2007.05.012","article-title":"Endogenous and synthetic neurosteroids in treatment of Niemann\u2013Pick Type C disease","volume":"57","author":"Mellon","year":"2008","journal-title":"Brain Res Rev"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0140","doi-asserted-by":"crossref","first-page":"1321","DOI":"10.1086\/302626","article-title":"Niemann\u2013Pick C1 disease: the I1061T substitution is a frequent mutant allele in patients of Western European descent and correlates with a classic juvenile phenotype","volume":"65","author":"Millat","year":"1999","journal-title":"Am J Hum Genet"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0145","doi-asserted-by":"crossref","first-page":"47","DOI":"10.1016\/0165-0270(84)90007-4","article-title":"Developments of a water-maze procedure for studying spatial learning in the rat","volume":"11","author":"Morris","year":"1984","journal-title":"J Neurosci Methods"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0150","doi-asserted-by":"crossref","first-page":"9627","DOI":"10.1074\/jbc.274.14.9627","article-title":"The Niemann\u2013Pick C1 protein resides in a vesicular compartment linked to retrograde transport of multiple lysosomal cargo","volume":"274","author":"Neufeld","year":"1999","journal-title":"J Biol Chem"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0155","doi-asserted-by":"crossref","first-page":"301","DOI":"10.1097\/01.nrl.0000094627.78754.5b","article-title":"A riddle wrapped in a mystery: understanding Niemann\u2013Pick disease, type C","volume":"9","author":"Patterson","year":"2003","journal-title":"Neurologist"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0160","doi-asserted-by":"crossref","first-page":"63","DOI":"10.1016\/j.bbalip.2004.08.012","article-title":"Before the loss: neuronal dysfunction in Niemann\u2013Pick type C disease","volume":"1685","author":"Paul","year":"2004","journal-title":"Biochim Biophys Acta"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0165","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\u2013Pick C1 (NPC1)-deficient mice","volume":"287","author":"Peake","year":"2012","journal-title":"J Biol Chem"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0170","doi-asserted-by":"crossref","first-page":"88","DOI":"10.1111\/j.1651-2227.2008.00656.x","article-title":"Substrate reduction therapy","volume":"97","author":"Platt","year":"2008","journal-title":"Acta Paediatr"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0175","doi-asserted-by":"crossref","first-page":"439","DOI":"10.1016\/j.ijdevneu.2012.07.002","article-title":"Oxidative stress in Niemann\u2013Pick type C patients: a protective role of N-butyl-deoxynojirimycin therapy","volume":"30","author":"Ribas","year":"2012","journal-title":"Int J Dev Neurosci"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0180","doi-asserted-by":"crossref","first-page":"279","DOI":"10.1002\/cne.10522","article-title":"Patterned Purkinje cell degeneration in mouse models of Niemann\u2013Pick type C disease","volume":"456","author":"Sarna","year":"2003","journal-title":"J Comp Neurol"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0185","doi-asserted-by":"crossref","first-page":"1033","DOI":"10.3727\/096368910X545086","article-title":"Human umbilical cord blood-derived mesenchymal stem cells protect against neuronal cell death and ameliorate motor deficits in Niemann Pick type C1 mice","volume":"20","author":"Seo","year":"2011","journal-title":"Cell Transplant"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0190","doi-asserted-by":"crossref","first-page":"446","DOI":"10.1002\/ajmg.a.32642","article-title":"The clinical spectrum of fetal Niemann\u2013Pick type C","volume":"149A","author":"Spiegel","year":"2009","journal-title":"Am J Med Genet A"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0195","doi-asserted-by":"crossref","first-page":"195","DOI":"10.1111\/j.1750-3639.1998.tb00145.x","article-title":"Mouse models of human lysosomal diseases","volume":"8","author":"Suzuki","year":"1998","journal-title":"Brain Pathol"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0200","doi-asserted-by":"crossref","first-page":"2331","DOI":"10.1194\/jlr.M028241","article-title":"Cyclodextrin mediates rapid changes in lipid balance in Npc1\u2212\/\u2212 mice without carrying cholesterol through the bloodstream","volume":"53","author":"Taylor","year":"2012","journal-title":"J Lipid Res"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0205","doi-asserted-by":"crossref","first-page":"16","DOI":"10.1186\/1750-1172-5-16","article-title":"Niemann\u2013Pick disease type C","volume":"5","author":"Vanier","year":"2010","journal-title":"Orphanet J Rare Dis"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0210","doi-asserted-by":"crossref","first-page":"269","DOI":"10.1034\/j.1399-0004.2003.00147.x","article-title":"Niemann\u2013Pick disease type C","volume":"64","author":"Vanier","year":"2003","journal-title":"Clin Genet"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0215","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/S0166-4328(01)00380-1","article-title":"Cognitive deficit and development of motor impairment in a mouse model of Niemann\u2013Pick type C disease","volume":"132","author":"V\u00f5ikar","year":"2002","journal-title":"Behav Brain Res"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0220","doi-asserted-by":"crossref","first-page":"495","DOI":"10.1016\/S0091-6773(76)90901-9","article-title":"Open-field behavior in muroid rodents","volume":"17","author":"Wilson","year":"1976","journal-title":"Behav Biol"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0225","doi-asserted-by":"crossref","first-page":"351","DOI":"10.1016\/j.ymgme.2009.12.006","article-title":"Miglustat in adult and juvenile patients with Niemann\u2013Pick disease type C: long-term data from a clinical trial","volume":"99","author":"Wraith","year":"2010","journal-title":"Mol Genet Metab"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0230","doi-asserted-by":"crossref","first-page":"49","DOI":"10.1093\/jnen\/60.1.49","article-title":"Neurons in Niemann\u2013Pick disease type C accumulate gangliosides as well as unesterified cholesterol and undergo dendritic and axonal alterations","volume":"60","author":"Zervas","year":"2001","journal-title":"J Neuropathol Exp Neurol"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0235","doi-asserted-by":"crossref","first-page":"1283","DOI":"10.1016\/S0960-9822(01)00396-7","article-title":"Critical role for glycosphingolipids in Niemann\u2013Pick disease type C","volume":"11","author":"Zervas","year":"2001","journal-title":"Curr Biol"},{"key":"10.1016\/j.neuroscience.2013.08.001_b0240","doi-asserted-by":"crossref","first-page":"212","DOI":"10.1002\/hipo.20741","article-title":"Decreased purinergic inhibition of synaptic activity in a mouse model of Niemann\u2013Pick disease type C","volume":"21","author":"Zhou","year":"2011","journal-title":"Hippocampus"}],"container-title":["Neuroscience"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0306452213006799?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0306452213006799?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2018,10,13]],"date-time":"2018-10-13T21:00:14Z","timestamp":1539464414000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0306452213006799"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2013,11]]},"references-count":48,"alternative-id":["S0306452213006799"],"URL":"https:\/\/doi.org\/10.1016\/j.neuroscience.2013.08.001","relation":{},"ISSN":["0306-4522"],"issn-type":[{"value":"0306-4522","type":"print"}],"subject":[],"published":{"date-parts":[[2013,11]]}}}