Effects of 6-O-α-maltosyl-β cyclodextrin on lipid metabolism in Npc1-deficient Chinese hamster ovary cells
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Carstea, 1997, Niemann-Pick C1 disease gene: homology to mediators of cholesterol homeostasis, Science., 277, 228, 10.1126/science.277.5323.228
Naureckiene, 2000, Identification of HE1 as the second gene of Niemann-Pick C disease, Science., 290, 2298, 10.1126/science.290.5500.2298
Vanier, 2010, Niemann-Pick disease type C, Orphanet J. Rare Dis., 5, 1, 10.1186/1750-1172-5-16
Bi, 2010, Cholesterol in Niemann-Pick Type C disease, Subcell. Biochem., 51, 319, 10.1007/978-90-481-8622-8_11
Vance, 2014, Niemann-Pick C disease and mobilization of lysosomal cholesterol by cyclodextrin, J. Lipid Res., 55, 1609, 10.1194/jlr.R047837
Kwon, 2009, Structure of N-terminal domain of npc1 reveals distinct subdomains for binding and transfer of cholesterol, Cell., 137, 1213, 10.1016/j.cell.2009.03.049
Wang, 2010, Identification of surface residues on Niemann-pick C2 essential for hydrophobic handoff of cholesterol to NPC1 in lysosomes, Cell Metab., 12, 166, 10.1016/j.cmet.2010.05.016
Matsuo, 2013, Effects of cyclodextrin in two patients with Niemann-Pick Type C disease, Mol. Genet. Metab., 108, 76, 10.1016/j.ymgme.2012.11.005
Megías-Vericat, 2017, Early experience with compassionate use of 2 hydroxypropyl-beta-cyclodextrin for Niemann-Pick type C disease: review of initial published cases, Neurol. Sci., 38, 727, 10.1007/s10072-017-2833-9
Hastings, 2019, Expanded access with intravenous hydroxypropyl-β-cyclodextrin to treat children and young adults with Niemann-Pick disease type C1: a case report analysis, Orphanet J. Rare Dis., 14, 228, 10.1186/s13023-019-1207-1
Tanaka, 2015, Efficacy of 2-hydroxypropyl-β-cyclodextrin in Niemann-Pick disease type C model mice and its pharmacokinetic analysis in a patient with the disease, Biol. Pharm. Bull., 38, 844, 10.1248/bpb.b14-00726
Camargo, 2001, Cyclodextrins in the treatment of a mouse model of Niemann-Pick C disease, Life Sci., 70, 131, 10.1016/S0024-3205(01)01384-4
Liu, 2009, Reversal of defective lysosomal transport in NPC disease ameliorates liver dysfunction and neurodegeneration in the npc1−/− mouse, Proc. Natl. Acad. Sci. U. S. A., 106, 2377, 10.1073/pnas.0810895106
Peake, 2012, Normalization of cholesterol homeostasis by 2-hydroxypropyl-β-cyclodextrin in neurons and glia from Niemann-Pick C1 (NPC1)-deficient mice, J. Biol. Chem., 287, 9290, 10.1074/jbc.M111.326405
Liu, 2012, Therapeutic potential of cyclodextrins in the treatment of Niemann-Pick type C disease, Clin. Lipidol., 7, 289, 10.2217/clp.12.31
Yergey, 2017, Characterization of hydroxypropyl-β-cyclodextrins used in the treatment of Niemann-Pick disease type C1, PLoS One, 12, 10.1371/journal.pone.0175478
Chien, 2013, Lung toxicity of hydroxypropyl-β-cyclodextrin infusion, Mol. Genet. Metab., 109, 231, 10.1016/j.ymgme.2013.04.003
Davidson, 2016, Efficacy and ototoxicity of different cyclodextrins in Niemann-Pick C disease, Ann. Clin. Transl. Neurol., 3, 366, 10.1002/acn3.306
Ward, 2010, 2-hydroxypropyl-β-cyclodextrin raises hearing threshold in normal cats and in cats with Niemann-Pick type C disease, Pediatr. Res., 68, 52, 10.1203/PDR.0b013e3181df4623
Okada, 1988, Some properties and the inclusion behavior of branched cyclodextrins, Chem. Pharm. Bull. (Tokyo), 36, 2176, 10.1248/cpb.36.2176
Okada, 2012, Effect of 6-O-α-maltosyl-β cyclodextrin and its cholesterol inclusion complex on cellular cholesterol levels and ABCA1 and ABCG1 expression in mouse mastocytoma P-815 cells, Carbohydr. Res., 357, 68, 10.1016/j.carres.2012.04.019
Yasmin, 2019, In vitro and in vivo evaluation of 6-O-α-maltosyl-β-cyclodextrin as a potential therapeutic agent against Niemann-Pick disease Type C, Int. J. Mol. Sci., 20, 1152, 10.3390/ijms20051152
Okada, 2018, Role of 6- O-α-maltosyl-β cyclodextrin in lysosomal cholesterol deprivation in Npc1-deficient Chinese hamster ovary cells, Carbohydr. Res., 455, 54, 10.1016/j.carres.2017.11.003
Chang, 2006, Cholesterol sensing, trafficking, and esterification, Annu. Rev. Cell Dev. Biol., 22, 129, 10.1146/annurev.cellbio.22.010305.104656
Wakil, 2009, Fatty acid metabolism: target for metabolic syndrome, J. Lipid Res. Apr., 50, S138, 10.1194/jlr.R800079-JLR200
Koike, 1998, Decreased membrane fluidity and unsaturated fatty acids in Niemann–Pick disease type C fibroblasts, Biochim. Biophys. Acta, 1406, 327, 10.1016/S0925-4439(98)00019-2
Passeggio, 2005, Flux of fatty acids through NPC1 lysosomes, J. Biol. Chem., 280, 10333, 10.1074/jbc.M413657200
Higaki, 2001, Isolation of NPC1-deficient Chinese hamster ovary cell mutants by gene trap mutagenesis, J. Biochem., 129, 875, 10.1093/oxfordjournals.jbchem.a002932
Nagata, 2001, Three-dimensional high voltage electron microscopy of thick biological specimens, Micron., 32, 387, 10.1016/S0968-4328(00)00005-6
Kakavanos, 2006, Stabilising normal and mis-sense variant α-glucosidase, FEBS Lett., 580, 4365, 10.1016/j.febslet.2006.06.096
Rosenbaum, 2010, Endocytosis of beta-cyclodextrins is responsible for cholesterol reduction in Niemann-Pick type C mutant cells, Proc. Natl. Acad. Sci. U. S. A., 107, 5477, 10.1073/pnas.0914309107
Rosenbaum, 2011, Niemann-Pick type C disease: molecular mechanisms and potential therapeutic approaches, J. Neurochem., 116, 789, 10.1111/j.1471-4159.2010.06976.x
Ramirez, 2011, Quantitative role of LAL, NPC2, and NPC1 in lysosomal cholesterol processing defined by genetic and pharmacological manipulations, J. Lipid Res., 52, 688, 10.1194/jlr.M013789
Brown, 1975, Receptor-dependent hydrolysis of cholesteryl esters contained in plasma low density lipoprotein, Proc. Natl. Acad. Sci. U. S. A., 72, 2925, 10.1073/pnas.72.8.2925
Goldstein, 1977, The low-density lipoprotein pathway and its relation to atherosclerosis, Annu. Rev. Biochem., 46, 897, 10.1146/annurev.bi.46.070177.004341
Groener, 1996, Metabolic fate of oleic acid derived from lysosomal degradation of cholesteryl oleate in human fibroblasts, J. Lipid Res., 37, 2271, 10.1016/S0022-2275(20)37475-7
Jaureguiberry, 2010, Membrane organization and regulation of cellular cholesterol homeostasis, J. Membr. Biol., 234, 183, 10.1007/s00232-010-9245-6
Howe, 2016, Cholesterol homeostasis: how do cells sense sterol excess?, Chem. Phys. Lipids, 199, 170, 10.1016/j.chemphyslip.2016.02.011
Litvinov, 2018, Intracellular and plasma membrane events in cholesterol transport and homeostasis, J. Lipids, 2018, 10.1155/2018/3965054
Dietschy, 2004, Thematic review series: brain lipids. Cholesterol metabolism in the central nervous system during early development and in the mature animal, J. Lipid Res., 45, 1375, 10.1194/jlr.R400004-JLR200
Soccio, 2004, Intracellular cholesterol transport, Arterioscler. Thromb. Vasc. Biol., 24, 1150, 10.1161/01.ATV.0000131264.66417.d5
Ouimet, 2012, Regulation of lipid droplet cholesterol efflux from macrophage foam cells, Arterioscler. Thromb. Vasc. Biol., 32, 575, 10.1161/ATVBAHA.111.240705
Frolov, 2003, NPC1 and NPC2 regulate cellular cholesterol homeostasis through generation of low density lipoprotein cholesterol-derived oxysterols, J. Biol. Chem., 278, 25517, 10.1074/jbc.M302588200
Lange, 2009, Regulation of fibroblast mitochondrial 27-hydroxychoresterol production by active plasma membrane cholesterol, J. Lipid Res., 50, 1881, 10.1194/jlr.M900116-JLR200
Roberg-Larsen, 2012, High sensitivity measurements of active oxysterols with automated filtration/filter backflush-solid phase extraction-liquid chromatography-mass spectrometry, J. Chromatogr. A, 1255, 291, 10.1016/j.chroma.2012.02.002
Chang, 1997, Acyl-coenzyme A: cholesterol acyltransferase, Annu. Rev. Biochem., 66, 613, 10.1146/annurev.biochem.66.1.613
Wang, 2017, Cholesterol and fatty acids regulate cysteine ubiquitylation of ACAT2 through competitive oxidation, Nat. Cell Biol., 19, 808, 10.1038/ncb3551
Byers, 1989, Defective activity of acyl-CoA:cholesterol O-acyltransferase in Niemann-Pick type C and type D fibroblasts, Biochem. J., 262, 713, 10.1042/bj2620713
Sané, 2006, Localization and role of NPC1L1 in cholesterol absorption in human intestine, J. Lipid Res., 47, 2112, 10.1194/jlr.M600174-JLR200
Mazière, 1986, Changes in the biosynthesis, esterification and efflux of cholesterol in fibroblasts in culture from patients with Niemann-Pick disease type C, C. R. Seances Soc. Biol. Fil., 180, 669
Kamikawa, 2014, ACAT1-associated late endosomes/lysosomes significantly improve impaired intracellular cholesterol metabolism and the survival of Niemann-pick Type C mice, Acta Histochem. Cytochem., 47, 35, 10.1267/ahc.13033
Ebner, 2018, Evaluation of two liver treatment strategies in a mouse model of Niemann-Pick-disease type C1, Int. J. Mol. Sci., 19, 972, 10.3390/ijms19040972
Xie, 1999, Cholesterol accumulation in tissues of the Niemann-pick type C mouse is determined by the rate of lipoprotein-cholesterol uptake through the coated-pit pathway in each organ, Proc. Natl. Acad. Sci. U. S. A., 96, 11992, 10.1073/pnas.96.21.11992
Walter, 2009, Endosomal lipid accumulation in NPC1 leads to inhibition of PKC, hypophosphorylation of vimentin and Rab9 entrapment, Biol. Cell., 101, 141, 10.1042/BC20070171
Tamari, 2013, PKC activation in Niemann pick C1 cells restores subcellular cholesterol transport, PLoS One, 8, 10.1371/journal.pone.0074169
Yan, 2015, Long-chain acyl CoA synthetase in fatty acid metabolism involved in liver and other diseases: an update, World J. Gastroenterol., 21, 3492, 10.3748/wjg.v21.i12.3492
Matsuzaka, 2009, Elovl6: a new player in fatty acid metabolism and insulin sensitivity, J. Mol. Med. (Berl), 87, 379, 10.1007/s00109-009-0449-0
Green, 2011, Modulation of palmitate-induced endoplasmic reticulum stress and apoptosis in pancreatic β-cells by stearoyl-CoA desaturase and Elovl6, Am. J. Physiol. Endocrinol. Metab., 300, E640, 10.1152/ajpendo.00544.2010
Ntambi, 1995, The regulation of stearoyl-CoA desaturase (SCD), Prog. Lipid Res., 34, 139, 10.1016/0163-7827(94)00010-J