Effects of 6-O-α-maltosyl-β cyclodextrin on lipid metabolism in Npc1-deficient Chinese hamster ovary cells

Molecular Genetics and Metabolism - Tập 137 - Trang 239-248 - 2022
By Yasuyo Okada1, Sayako Kuroiwa1, Ayaka Noi1, Ayaka Tanaka1, Junichi Nishikawa1, Yuki Kondo2, Yoichi Ishitsuka2, Tetsumi Irie3, Katsumi Higaki4, Muneaki Matsuo5, Atsushi Ichikawa1,6
1School of Pharmacy and Pharmaceutical Sciences, Mukogawa Women's University, 11-68 Kyuban-cho, Koshien, Nishinomiya, Hyogo 663-8179, Japan
2Department of Clinical Chemistry and Informatics, Graduate School of Pharmaceutical Sciences, Kumamoto University, 5-1 Oe-honmachi, Chuo-ku, Kumamoto 862-0973, Japan
3Department of Pharmaceutical Packaging Technology, Faculty of Life Sciences, Kumamoto University, 5-1 Oe-honmachi, Chuo-ku, Kumamoto 862-0973, Japan
4Research Initiative Center, Organization for Research Initiative and Promotion, Tottori University, 86 Nishi-cho, Yonago 683-8503, Japan
5Department of Pediatrics, Faculty of Medicine, Saga University, 5-1-1 Nabeshima, Saga 849-8501, Japan
6Bio-Education Laboratory, Tawara Building #702, 1-21-33 Higashinakajima, Osaka 533-0033, Japan

Tài liệu tham khảo

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