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Therapeutic potential of cyclodextrins in the treatment of Niemann–Pick type C disease

Pages 289-301 | Published online: 18 Jan 2017

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Tatiana Bremova-Ertl & Susanne Schneider. (2023) Current advancements in therapy for Niemann-Pick disease: progress and pitfalls. Expert Opinion on Pharmacotherapy 24:11, pages 1229-1247.
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Veronica Antipova, Lisa-Marie Steinhoff, Carsten Holzmann, Arndt Rolfs, Carlos Junior Hempel, Martin Witt & Andreas Wree. (2022) Organ Weights in NPC1 Mutant Mice Partly Normalized by Various Pharmacological Treatment Approaches. International Journal of Molecular Sciences 24:1, pages 573.
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Caroline Hastings, Benny Liu, Bryan Hurst, Gerald F. Cox & Sharon Hrynkow. (2022) Intravenous 2-hydroxypropyl-β-cyclodextrin (Trappsol® Cyclo™) demonstrates biological activity and impacts cholesterol metabolism in the central nervous system and peripheral tissues in adult subjects with Niemann-Pick Disease Type C1: Results of a phase 1 trial. Molecular Genetics and Metabolism 137:4, pages 309-319.
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Victoria N. Gorbunova & Natalia V. Buchinskaia. (2022) Lysosomal storage diseases. Sphingolipidoses — sphingomyelin lipidosis, or Niemann–Pick disease, Wolman disease. Pediatrician (St. Petersburg) 13:4, pages 5-27.
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By Yasuyo Okada, Sayako Kuroiwa, Ayaka Noi, Ayaka Tanaka, Junichi Nishikawa, Yuki Kondo, Yoichi Ishitsuka, Tetsumi Irie, Katsumi Higaki, Muneaki Matsuo & Atsushi Ichikawa. (2022) Effects of 6-O-α-maltosyl-β cyclodextrin on lipid metabolism in Npc1-deficient Chinese hamster ovary cells. Molecular Genetics and Metabolism 137:3, pages 239-248.
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Sahin Demirci, Dawin Khiev, Mehmet Can, Mehtap Sahiner, Manas R. Biswal, Ramesh S. Ayyala & Nurettin Sahiner. (2021) Chemically Cross-Linked Poly(β-Cyclodextrin) Particles as Promising Drug Delivery Materials. ACS Applied Polymer Materials 3:12, pages 6238-6251.
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Anika V. Prabhu, Insung Kang, Raffaella De Pace, Christopher A. Wassif, Hideji Fujiwara, Pamela Kell, Xuntian Jiang, Daniel S. Ory, Juan S. Bonifacino, Michael E. Ward & Forbes D. Porter. (2021) A human iPSC-derived inducible neuronal model of Niemann-Pick disease, type C1. BMC Biology 19:1.
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Antonino Puglisi, Simone Bassini & Erik Reimhult. (2021) Cyclodextrin-Appended Superparamagnetic Iron Oxide Nanoparticles as Cholesterol-Mopping Agents. Frontiers in Chemistry 9.
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Shreya S. Soni, Abdulrahman Alsasa & Christopher B. Rodell. (2021) Applications of Macrocyclic Host Molecules in Immune Modulation and Therapeutic Delivery. Frontiers in Chemistry 9.
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Charina M. Ramirez, Anna M. Taylor, Adam M. Lopez, Joyce J. Repa & Stephen D. Turley. (2020) Delineation of metabolic responses of Npc1 mice lacking the cholesterol-esterifying enzyme SOAT2 to acute treatment with 2-hydroxypropyl-β-cyclodextrin. Steroids 164, pages 108725.
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Deborah Moreira Rangel, Matias Carvalho Aguiar Melo, José Luiz Pedroso, Orlando Graziani Povoas Barsottini, Manoel Alves Sobreira-Neto & Pedro Braga-Neto. (2020) Beyond the Typical Syndrome: Understanding Non-motor Features in Niemann-Pick Type C Disease. The Cerebellum 19:5, pages 722-738.
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Audrey Thurm, Colby Chlebowski, Lisa Joseph, Cristan Farmer, Dee Adedipe, Madison Weiss, Edythe Wiggs, Nicole Farhat, Simona Bianconi, Elizabeth Berry-Kravis & Forbes D. Porter. (2020) Neurodevelopmental Characterization of Young Children Diagnosed with Niemann-Pick Disease, Type C1. Journal of Developmental & Behavioral Pediatrics 41:5, pages 388-396.
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Adrián Matencio, Silvia Navarro-Orcajada, Alejandro González-Ramón, Francisco García-Carmona & José Manuel López-Nicolás. (2020) Recent advances in the treatment of Niemann pick disease type C: A mini-review. International Journal of Pharmaceutics 584, pages 119440.
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Xiao Feng, Fan Yang, Michael Rabenstein, Zhen Wang, Moritz J Frech, Andreas Wree, Anja U Bräuer, Martin Witt, Anne Gläser, Andreas Hermann, Arndt Rolfs & Jiankai Luo. (2020) Stimulation of mGluR1/5 Improves Defective Internalization of AMPA Receptors in NPC1 Mutant Mouse. Cerebral Cortex 30:3, pages 1465-1480.
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Mino R. Caira. (2019) Cyclodextrin Inclusion of Medicinal Compounds for Enhancement of their Physicochemical and Biopharmaceutical Properties. Current Topics in Medicinal Chemistry 19:25, pages 2357-2370.
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Angelique M. L. Scantlebery, Peter Ochodnicky, Lotte Kors, Elena Rampanelli, Loes M. Butter, Chaima El Boumashouli, Nike Claessen, Gwen J. Teske, Marius A. van den Bergh Weerman, Jaklien C. Leemans, Joris J. T. H. Roelofs & Sandrine Florquin. (2019) β-Cyclodextrin counteracts obesity in Western diet-fed mice but elicits a nephrotoxic effect. Scientific Reports 9:1.
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A.C. Carreira, T.C. Santos, M.A. Lone, E. Zupančič, E. Lloyd-Evans, R.F.M. de Almeida, T. Hornemann & L.C. Silva. (2019) WITHDRAWN: Mammalian sphingoid bases: Biophysical, physiological and pathological properties. Progress in Lipid Research, pages 100995.
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István Hajdu, János Angyal, Dezső Szikra, István Kertész, Milo Malanga, Éva Fenyvesi, Lajos Szente, Miklós Vecsernyés, Ildikó Bácskay, Judit Váradi, Pálma Fehér, Zoltán Ujhelyi, Gábor Vasvári, Ágnes Rusznyák, György Trencsényi & Ferenc Fenyvesi. (2019) Radiochemical synthesis and preclinical evaluation of 68Ga-labeled NODAGA-hydroxypropyl-beta-cyclodextrin (68Ga-NODAGA-HPBCD). European Journal of Pharmaceutical Sciences 128, pages 202-208.
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Thomas Rego, Sarah Farrand, Anita M. Y. Goh, Dhamidhu Eratne, Wendy Kelso, Simone Mangelsdorf, Dennis Velakoulis & Mark Walterfang. (2019) Psychiatric and Cognitive Symptoms Associated with Niemann-Pick Type C Disease: Neurobiology and Management. CNS Drugs 33:2, pages 125-142.
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Yingjie Zhou, Satoe Takahashi, Kazuaki Homma, Chongwen Duan, Jason Zheng, Mary Ann Cheatham & Jing Zheng. (2018) The susceptibility of cochlear outer hair cells to cyclodextrin is not related to their electromotile activity. Acta Neuropathologica Communications 6:1.
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Adam M. Lopez, Ryan D. Jones, Joyce J. Repa & Stephen D. Turley. (2018) Niemann-Pick C1-deficient mice lacking sterol O -acyltransferase 2 have less hepatic cholesterol entrapment and improved liver function . American Journal of Physiology-Gastrointestinal and Liver Physiology 315:4, pages G454-G463.
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Elias Christoforides, Andreas Papaioannou & Kostas Bethanis. (2018) Crystal structure of the inclusion complex of cholesterol in β-cyclodextrin and molecular dynamics studies. Beilstein Journal of Organic Chemistry 14, pages 838-848.
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Vincent Geoghegan, Kirsty Stainton, Stephanie M. Rainey, Thomas H. Ant, Adam A. Dowle, Tony Larson, Svenja Hester, Philip D. Charles, Benjamin Thomas & Steven P. Sinkins. (2017) Perturbed cholesterol and vesicular trafficking associated with dengue blocking in Wolbachia-infected Aedes aegypti cells. Nature Communications 8:1.
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Jason Gaspar, Jacques Mathieu & Pedro Alvarez. (2017) 2-Hydroxypropyl-beta-cyclodextrin (HPβCD) reduces age-related lipofuscin accumulation through a cholesterol-associated pathway. Scientific Reports 7:1.
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Maria Roch Santed, Maria Josep Cabañas Poy, Mireia Del Toro Riera, Carme Cañete Ramírez, Aurora Fernández Polo & Susana Clemente Bautista. (2017) Intrathecal cyclodextrin in the treatment of Niemann-Pick disease type C. European Journal of Hospital Pharmacy 24:3, pages 185-188.
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Christopher J. Collins, Bradley P. Loren, Md Suhail Alam, Yawo Mondjinou, Joseph L. Skulsky, Cheyenne R. Chaplain, Kasturi Haldar & David H. Thompson. (2017) Pluronic based β-cyclodextrin polyrotaxanes for treatment of Niemann-Pick Type C disease. Scientific Reports 7:1.
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Anja Meyer, Andreas Wree, René Günther, Carsten Holzmann, Oliver Schmitt, Arndt Rolfs & Martin Witt. (2017) Increased Regenerative Capacity of the Olfactory Epithelium in Niemann–Pick Disease Type C1. International Journal of Molecular Sciences 18:4, pages 777.
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William R. H. Evans & Chris J. Hendriksz. (2018) Niemann-Pick type C disease – the tip of the iceberg? A review of neuropsychiatric presentation, diagnosis and treatment. BJPsych Bulletin 41:2, pages 109-114.
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Lajos Szente, Julianna Szemán & Tamás Sohajda. (2016) Analytical characterization of cyclodextrins: History, official methods and recommended new techniques. Journal of Pharmaceutical and Biomedical Analysis 130, pages 347-365.
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Jeff Rappaport, Rachel L. Manthe, Melani Solomon, Carmen Garnacho & Silvia Muro. (2016) A Comparative Study on the Alterations of Endocytic Pathways in Multiple Lysosomal Storage Disorders. Molecular Pharmaceutics 13:2, pages 357-368.
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Gesine Saher & Sina Kristin Stumpf. (2015) Cholesterol in myelin biogenesis and hypomyelinating disorders. Biochimica et Biophysica Acta (BBA) - Molecular and Cell Biology of Lipids 1851:8, pages 1083-1094.
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Rodrigo Sierpe, Erika Lang, Paul Jara, Ariel R. Guerrero, Boris Chornik, Marcelo J. Kogan & Nicolás Yutronic. (2015) Gold Nanoparticles Interacting with β-Cyclodextrin–Phenylethylamine Inclusion Complex: A Ternary System for Photothermal Drug Release. ACS Applied Materials & Interfaces 7:28, pages 15177-15188.
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Adam M Lopez, Sandi J Terpack, Kenneth S Posey, Benny Liu, Charina M Ramirez & Stephen D Turley. (2014) Systemic administration of 2-hydroxypropyl-β-cyclodextrin to symptomatic Npc1-deficient mice slows cholesterol sequestration in the major organs and improves liver function. Clinical and Experimental Pharmacology and Physiology 41:10, pages 780-787.
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Atsushi Tamura & Nobuhiko Yui. (2014) Lysosomal-specific Cholesterol Reduction by Biocleavable Polyrotaxanes for Ameliorating Niemann-Pick Type C Disease. Scientific Reports 4:1.
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Muneaki Matsuo, Koki Shraishi, Koki Wada, Yoichi Ishitsuka, Hirohito Doi, Miyuki Maeda, Tatsuhiro Mizoguchi, Junya Eto, Sakiko Mochinaga, Hidetoshi Arima & Tetsumi Irie. (2014) Effects of intracerebroventricular administration of 2-hydroxypropyl-β-cyclodextrin in a patient with Niemann–Pick Type C disease. Molecular Genetics and Metabolism Reports 1, pages 391-400.
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Jen-Chieh Chuang, Adam M. Lopez, Kenneth S. Posey & Stephen D. Turley. (2014) Ezetimibe markedly attenuates hepatic cholesterol accumulation and improves liver function in the lysosomal acid lipase-deficient mouse, a model for cholesteryl ester storage disease. Biochemical and Biophysical Research Communications 443:3, pages 1073-1077.
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Charina M. Ramirez, Adam M. Lopez, Lam Q. Le, Kenneth S. Posey, Arthur G. Weinberg & Stephen D. Turley. (2014) Ontogenic changes in lung cholesterol metabolism, lipid content, and histology in mice with Niemann–Pick type C disease. Biochimica et Biophysica Acta (BBA) - Molecular and Cell Biology of Lipids 1841:1, pages 54-61.
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Caroline Hastings, Benny Liu, Bryan Hurst, Gerald F. Cox & Sharon Hrynkow. (2022) Intravenous 2-Hydroxypropyl-Β-Cyclodextrin (Trappsol® Cyclo™) Demonstrates Biological Activity and Impacts Cholesterol Metabolism in the Central Nervous System and Peripheral Tissues in Adult Subjects with Niemann-Pick Disease Type C1: Results of a Phase 1 Trial. SSRN Electronic Journal.
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