{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,6]],"date-time":"2026-10-06T23:32:39Z","timestamp":1791329559671,"version":"4.3.1"},"reference-count":25,"publisher":"Pharmaceutical Society of Japan","issue":"6","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Biological & Pharmaceutical Bulletin","Biol. Pharm. Bull."],"published-print":{"date-parts":[[2015]]},"DOI":"10.1248\/bpb.b14-00726","type":"journal-article","created":{"date-parts":[[2015,5,31]],"date-time":"2015-05-31T18:04:28Z","timestamp":1433095468000},"page":"844-851","source":"Crossref","is-referenced-by-count":88,"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"],"prefix":"10.1248","volume":"38","author":[{"given":"Yuta","family":"Tanaka","sequence":"first","affiliation":[{"name":"Department of Clinical Chemistry and Informatics, Graduate School of Pharmaceutical Sciences, Kumamoto University"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yusei","family":"Yamada","sequence":"additional","affiliation":[{"name":"Department of Clinical Chemistry and Informatics, Graduate School of Pharmaceutical Sciences, Kumamoto University"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yoichi","family":"Ishitsuka","sequence":"additional","affiliation":[{"name":"Department of Clinical Chemistry and Informatics, Graduate School of Pharmaceutical Sciences, Kumamoto University"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Muneaki","family":"Matsuo","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Faculty of Medicine, Saga University"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Koki","family":"Shiraishi","sequence":"additional","affiliation":[{"name":"Department of Clinical Chemistry and Informatics, Graduate School of Pharmaceutical Sciences, Kumamoto University"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Koki","family":"Wada","sequence":"additional","affiliation":[{"name":"Research Institute, Nihon Shokuhin Kako Co., Ltd."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yushiro","family":"Uchio","sequence":"additional","affiliation":[{"name":"Department of Clinical Chemistry and Informatics, Graduate School of Pharmaceutical Sciences, Kumamoto University"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yuki","family":"Kondo","sequence":"additional","affiliation":[{"name":"Department of Clinical Chemistry and Informatics, Graduate School of Pharmaceutical Sciences, Kumamoto University"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Toru","family":"Takeo","sequence":"additional","affiliation":[{"name":"Division of Reproductive Engineering, Center for Animal Resources and Development (CARD), Kumamoto University"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Naomi","family":"Nakagata","sequence":"additional","affiliation":[{"name":"Division of Reproductive Engineering, Center for Animal Resources and Development (CARD), Kumamoto University"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Taishi","family":"Higashi","sequence":"additional","affiliation":[{"name":"Department of Physical Pharmaceutics, Graduate School of Pharmaceutical Sciences, Kumamoto University"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Keiichi","family":"Motoyama","sequence":"additional","affiliation":[{"name":"Department of Physical Pharmaceutics, Graduate School of Pharmaceutical Sciences, Kumamoto University"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hidetoshi","family":"Arima","sequence":"additional","affiliation":[{"name":"Department of Physical Pharmaceutics, Graduate School of Pharmaceutical Sciences, Kumamoto University"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sakiko","family":"Mochinaga","sequence":"additional","affiliation":[{"name":"Department of Pharmacy, Faculty of Medicine, Saga University"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Katsumi","family":"Higaki","sequence":"additional","affiliation":[{"name":"Division of Functional Genomics, Research Center for Bioscience and Technology, Faculty of Medicine, Tottori University"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kousaku","family":"Ohno","sequence":"additional","affiliation":[{"name":"Sanin Rosai Hospital"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tetsumi","family":"Irie","sequence":"additional","affiliation":[{"name":"Department of Clinical Chemistry and Informatics, Graduate School of Pharmaceutical Sciences, Kumamoto University"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"163","reference":[{"key":"1","unstructured":"1) Carstea ED, Morris JA, Coleman KG, Loftus SK, Zhang D, Cummings C, Gu J, Rosenfeld MA, Pavan WJ, Krizman DB, Nagle J, Polymeropoulos MH, Sturley SL, Ioannou YA, Higgins ME, Comly M, Cooney A, Brown A, Kaneski CR, Blanchette-Mackie EJ, Dwyer NK, Neufeld EB, Chang TY, Liscum L, Strauss JF 3rd, Ohno K, Zeigler M, Carmi R, Sokol J, Markie D, O'Neill RR, van Diggelen OP, Elleder M, Patterson MC, Brady RO, Vanier MT, Pentchev PG, Tagle DA. Niemann\u2013Pick C1 disease gene: homology to mediators of cholesterol homeostasis. Science<\/i>, 277<\/b>, 228\u2013231 (1997)."},{"key":"2","doi-asserted-by":"crossref","unstructured":"2) Naureckiene S, Sleat DE, Lackland H, Fensom A, Vanier MT, Wattiaux R, Jadot M, Lobel P. Identification of HE1 as the second gene of Niemann\u2013Pick C disease. Science<\/i>, 290<\/b>, 2298\u20132301 (2000).","DOI":"10.1126\/science.290.5500.2298"},{"key":"3","doi-asserted-by":"crossref","unstructured":"3) Vanier MT. Niemann\u2013Pick disease type C. Orphanet J. Rare Dis.<\/i>, 5<\/b>, 16 (2010).","DOI":"10.1186\/1750-1172-5-16"},{"key":"4","doi-asserted-by":"crossref","unstructured":"4) Matsuo M, Togawa M, Hirabaru K, Mochinaga S, Narita A, Adachi M, Egashira M, Irie T, Ohno K. Effects of cyclodextrin in two patients with Niemann\u2013Pick type C disease. Mol. Genet. Metab.<\/i>, 108<\/b>, 76\u201381 (2013).","DOI":"10.1016\/j.ymgme.2012.11.005"},{"key":"5","doi-asserted-by":"crossref","unstructured":"5) Wojtanik KM, Liscum L. The transport of low density lipoprotein-derived cholesterol to the plasma membrane is defective in NPC1 cells. J. Biol. Chem.<\/i>, 278<\/b>, 14850\u201314856 (2003).","DOI":"10.1074\/jbc.M300488200"},{"key":"6","doi-asserted-by":"crossref","unstructured":"6) Peake KB, Vance JE. Normalization of cholesterol homeostasis by 2-hydroxypropyl-beta-cyclodextrin in neurons and glia from Niemann\u2013Pick C1 (NPC1)-deficient mice. J. Biol. Chem.<\/i>, 287<\/b>, 9290\u20139298 (2012).","DOI":"10.1074\/jbc.M111.326405"},{"key":"7","doi-asserted-by":"crossref","unstructured":"7) Liu B, Turley SD, Burns DK, Miller AM, Repa JJ, Dietschy JM. Reversal of defective lysosomal transport in NPC disease ameliorates liver dysfunction and neurodegeneration in the npc1\u2212\/\u2212<\/sup> mouse. Proc. Natl. Acad. Sci. U.S.A.<\/i>, 106<\/b>, 2377\u20132382 (2009).","DOI":"10.1073\/pnas.0810895106"},{"key":"8","doi-asserted-by":"crossref","unstructured":"8) Davidson CD, Ali NF, Micsenyi MC, Stephney G, Renault S, Dobrenis K, Ory DS, Vanier MT, Walkley SU. Chronic cyclodextrin treatment of murine Niemann\u2013Pick C disease ameliorates neuronal cholesterol and glycosphingolipid storage and disease progression. PLoS ONE<\/i>, 4<\/b>, e6951 (2009).","DOI":"10.1371\/journal.pone.0006951"},{"key":"9","doi-asserted-by":"crossref","unstructured":"9) Ramirez CM, Liu B, Taylor AM, Repa JJ, Burns DK, Weinberg AG, Turley SD, Dietschy JM. Weekly cyclodextrin administration normalizes cholesterol metabolism in nearly every organ of the Niemann\u2013Pick type C1 mouse and markedly prolongs life. Pediatr. Res.<\/i>, 68<\/b>, 309\u2013315 (2010).","DOI":"10.1203\/PDR.0b013e3181ee4dd2"},{"key":"10","doi-asserted-by":"crossref","unstructured":"10) Yu D, Swaroop M, Wang M, Baxa U, Yang R, Yan Y, Coksaygan T, DeTolla L, Marugan JJ, Austin CP, McKew JC, Gong DW, Zheng W. Niemann\u2013Pick disease type c: induced pluripotent stem cell-derived neuronal cells for modeling neural disease and evaluating drug efficacy. J. Biomol. Screen.<\/i>, 19<\/b>, 1164\u20131173 (2014).","DOI":"10.1177\/1087057114537378"},{"key":"11","unstructured":"11) Loftus SK, Morris JA, Carstea ED, Gu JZ, Cummings C, Brown A, Ellison J, Ohno K, Rosenfeld MA, Tagle DA, Pentchev PG, Pavan WJ. Murine model of Niemann\u2013Pick C disease: mutation in a cholesterol homeostasis gene. Science<\/i>, 277<\/b>, 232\u2013235 (1997)."},{"key":"12","doi-asserted-by":"crossref","unstructured":"12) Tanaka Y, Ishitsuka Y, Yamada Y, Kondo Y, Takeo T, Nakagata N, Higashi T, Motoyama K, Arima H, Matsuo M, Higaki K, Ohno K, Irie T. Influence of Npc1<\/i> genotype on the toxicity of hydroxypropyl-\u03b2-cyclodextrin, a potentially therapeutic agent, in Niemann\u2013Pick type C disease models. Mol. Genet. Metab. Rep.<\/i>, 1<\/b>, 19\u201330 (2014).","DOI":"10.1016\/j.ymgmr.2013.12.003"},{"key":"13","doi-asserted-by":"crossref","unstructured":"13) Frijlink HW, Visser J, Drenth BF. Determination of cyclodextrins in biological fluids by high-performance liquid chromatography with negative colorimetric detection using post-column complexation with phenolphthalein. J. Chromatogr.<\/i>, 415<\/b>, 325\u2013333 (1987).","DOI":"10.1016\/S0378-4347(00)83224-7"},{"key":"14","doi-asserted-by":"crossref","unstructured":"14) Szathmary SC. Determination of hydroxypropyl-beta-cyclodextrin in plasma and urine by size-exclusion chromatography with post-column complexation. J. Chromatogr.<\/i>, 487<\/b>, 99\u2013105 (1989).","DOI":"10.1016\/S0378-4347(00)83011-X"},{"key":"15","doi-asserted-by":"crossref","unstructured":"15) Aqul A, Liu B, Ramirez CM, Pieper AA, Estill SJ, Burns DK, Liu B, Repa JJ, Turley SD, Dietschy JM. Unesterified cholesterol accumulation in late endosomes\/lysosomes causes neurodegeneration and is prevented by driving cholesterol export from this compartment. J. Neurosci.<\/i>, 31<\/b>, 9404\u20139413 (2011).","DOI":"10.1523\/JNEUROSCI.1317-11.2011"},{"key":"16","doi-asserted-by":"crossref","unstructured":"16) Tamura A, Yui N. Lysosomal-specific cholesterol reduction by biocleavable polyrotaxanes for ameliorating Niemann\u2013Pick type C disease. Sci. Rep.<\/i>, 4<\/b>, 4356 (2014).","DOI":"10.1038\/srep04356"},{"key":"17","doi-asserted-by":"crossref","unstructured":"17) Lopez AM, Terpack SJ, Posey KS, Liu B, Ramirez CM, Turley SD. Systemic administration of 2-hydroxypropyl-\u03b2-cyclodextrin to symptomatic Npc1-deficient mice slows cholesterol sequestration in the major organs and improves liver function. Clin. Exp. Pharmacol. Physiol.<\/i>, 41<\/b>, 780\u2013787 (2014).","DOI":"10.1111\/1440-1681.12285"},{"key":"18","doi-asserted-by":"crossref","unstructured":"18) Frijlink HW, Visser J, Hefting NR, Oosting R, Meijer DK, Lerk CF. The pharmacokinetics of beta-cyclodextrin and hydroxypropyl-beta-cyclodextrin in the rat. Pharm. Res.<\/i>, 7<\/b>, 1248\u20131252 (1990).","DOI":"10.1023\/A:1015929720063"},{"key":"19","doi-asserted-by":"crossref","unstructured":"19) Irie T, Uekama K. Pharmaceutical applications of cyclodextrins. III. Toxicological issues and safety evaluation. J. Pharm. Sci.<\/i>, 86<\/b>, 147\u2013162 (1997).","DOI":"10.1021\/js960213f"},{"key":"20","doi-asserted-by":"crossref","unstructured":"20) Rosenbaum AI, Zhang G, Warren JD, Maxfield FR. Endocytosis of beta-cyclodextrins is responsible for cholesterol reduction in Niemann\u2013Pick type C mutant cells. Proc. Natl. Acad. Sci. U.S.A.<\/i>, 107<\/b>, 5477\u20135482 (2010).","DOI":"10.1073\/pnas.0914309107"},{"key":"21","doi-asserted-by":"crossref","unstructured":"21) Camargo F, Erickson RP, Garver WS, Hossain GS, Carbone PN, Heidenreich RA, Blanchard J. Cyclodextrins in the treatment of a mouse model of Niemann\u2013Pick C disease. Life Sci.<\/i>, 70<\/b>, 131\u2013142 (2001).","DOI":"10.1016\/S0024-3205(01)01384-4"},{"key":"22","doi-asserted-by":"crossref","unstructured":"22) Liu B, Ramirez CM, Miller AM, Repa JJ, Turley SD, Dietschy JM. Cyclodextrin overcomes the transport defect in nearly every organ of NPC1 mice leading to excretion of sequestered cholesterol as bile acid. J. Lipid Res.<\/i>, 51<\/b>, 933\u2013944 (2010).","DOI":"10.1194\/jlr.M000257"},{"key":"23","doi-asserted-by":"crossref","unstructured":"23) Rosenbaum AI, Maxfield FR. Niemann\u2013Pick type C disease: molecular mechanisms and potential therapeutic approaches. J. Neurochem.<\/i>, 116<\/b>, 789\u2013795 (2011).","DOI":"10.1111\/j.1471-4159.2010.06976.x"},{"key":"24","doi-asserted-by":"crossref","unstructured":"24) Vance JE, Peake KB. Function of the Niemann\u2013Pick type C proteins and their bypass by cyclodextrin. Curr. Opin. Lipidol.<\/i>, 22<\/b>, 204\u2013209 (2011).","DOI":"10.1097\/MOL.0b013e3283453e69"},{"key":"25","doi-asserted-by":"crossref","unstructured":"25) Taylor AM, Liu B, Mari Y, Liu B, Repa JJ. Cyclodextrin mediates rapid changes in lipid balance in Npc1\u2212\/\u2212<\/sup> mice without carrying cholesterol through the bloodstream. J. Lipid Res.<\/i>, 53<\/b>, 2331\u20132342 (2012).","DOI":"10.1194\/jlr.M028241"}],"container-title":["Biological & Pharmaceutical Bulletin","Biological and Pharmaceutical Bulletin"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.jstage.jst.go.jp\/article\/bpb\/38\/6\/38_b14-00726\/_pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2019,8,25]],"date-time":"2019-08-25T17:56:27Z","timestamp":1566755787000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.jstage.jst.go.jp\/article\/bpb\/38\/6\/38_b14-00726\/_article"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2015]]},"references-count":25,"journal-issue":{"issue":"6","published-print":{"date-parts":[[2015]]}},"URL":"https:\/\/doi.org\/10.1248\/bpb.b14-00726","relation":{},"ISSN":["0918-6158","1347-5215"],"issn-type":[{"value":"0918-6158","type":"print"},{"value":"1347-5215","type":"electronic"}],"subject":[],"published":{"date-parts":[[2015]]}}}