Therapeutic potential of cyclodextrins in the treatment of Niemann–Pick type C disease
Published 2012 View Full Article
- Home
- Publications
- Publication Search
- Publication Details
Title
Therapeutic potential of cyclodextrins in the treatment of Niemann–Pick type C disease
Authors
Keywords
-
Journal
Clinical Lipidology
Volume 7, Issue 3, Pages 289-301
Publisher
Future Medicine Ltd
Online
2012-07-13
DOI
10.2217/clp.12.31
References
Ask authors/readers for more resources
Related references
Note: Only part of the references are listed.- Normalization of Cholesterol Homeostasis by 2-Hydroxypropyl-β-cyclodextrin in Neurons and Glia from Niemann-Pick C1 (NPC1)-deficient Mice
- (2012) Kyle B. Peake et al. JOURNAL OF BIOLOGICAL CHEMISTRY
- Sterol Transfer between Cyclodextrin and Membranes: Similar but Not Identical Mechanism to NPC2-Mediated Cholesterol Transfer
- (2011) Leslie A. McCauliff et al. BIOCHEMISTRY
- A novel mouse model of Niemann–Pick type C disease carrying a D1005G-Npc1 mutation comparable to commonly observed human mutations
- (2011) Robert A. Maue et al. HUMAN MOLECULAR GENETICS
- Quantitative role of LAL, NPC2, and NPC1 in lysosomal cholesterol processing defined by genetic and pharmacological manipulations
- (2011) Charina M. Ramirez et al. JOURNAL OF LIPID RESEARCH
- Unesterified Cholesterol Accumulation in Late Endosomes/Lysosomes Causes Neurodegeneration and Is Prevented by Driving Cholesterol Export from This Compartment
- (2011) A. Aqul et al. JOURNAL OF NEUROSCIENCE
- Pulmonary function and pathology in hydroxypropyl-beta-cyclodextin-treated and untreated Npc1−/− mice
- (2011) Akshay Muralidhar et al. MOLECULAR GENETICS AND METABOLISM
- Lack of efficacy of curcumin on neurodegeneration in the mouse model of Niemann–Pick C1
- (2011) Ivan A. Borbon et al. PHARMACOLOGY BIOCHEMISTRY AND BEHAVIOR
- Amino acid substitution in NPC1 that abolishes cholesterol binding reproduces phenotype of complete NPC1 deficiency in mice
- (2011) X. Xie et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Niemann-Pick type C 1 function requires lumenal domain residues that mediate cholesterol-dependent NPC2 binding
- (2011) M. S. Deffieu et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Niemann-Pick disease group C: clinical variability and diagnosis based on defective cholesterol esterification: A collaborative study on 70 patients
- (2010) Marie T. Vanier et al. CLINICAL GENETICS
- Niemann-Pick disease type C
- (2010) Marie T Vanier Orphanet Journal of Rare Diseases
- 2-Hydroxypropyl-β-Cyclodextrin Raises Hearing Threshold in Normal Cats and in Cats With Niemann-Pick Type C Disease
- (2010) Sarah Ward et al. PEDIATRIC RESEARCH
- Weekly Cyclodextrin Administration Normalizes Cholesterol Metabolism in Nearly Every Organ of the Niemann-Pick Type C1 Mouse and Markedly Prolongs Life
- (2010) Charina M Ramirez et al. PEDIATRIC RESEARCH
- Endocytosis of beta-cyclodextrins is responsible for cholesterol reduction in Niemann-Pick type C mutant cells
- (2010) A. I. Rosenbaum et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Structure of N-Terminal Domain of NPC1 Reveals Distinct Subdomains for Binding and Transfer of Cholesterol
- (2009) Hyock Joo Kwon et al. CELL
- Cyclodextrin overcomes the transport defect in nearly every organ of NPC1 mice leading to excretion of sequestered cholesterol as bile acid
- (2009) Benny Liu et al. JOURNAL OF LIPID RESEARCH
- Miglustat in patients with Niemann-Pick disease Type C (NP-C): A multicenter observational retrospective cohort study
- (2009) M. Pineda et al. MOLECULAR GENETICS AND METABOLISM
- Miglustat in adult and juvenile patients with Niemann–Pick disease type C: Long-term data from a clinical trial
- (2009) James E. Wraith et al. MOLECULAR GENETICS AND METABOLISM
- Chronic Cyclodextrin Treatment of Murine Niemann-Pick C Disease Ameliorates Neuronal Cholesterol and Glycosphingolipid Storage and Disease Progression
- (2009) Cristin D. Davidson et al. PLoS One
- Cyclodextrin overcomes deficient lysosome-to-endoplasmic reticulum transport of cholesterol in Niemann-Pick type C cells
- (2009) L. Abi-Mosleh et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Reversal of defective lysosomal transport in NPC disease ameliorates liver dysfunction and neurodegeneration in the npc1-/- mouse
- (2009) B. Liu et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Regulation of Sterol Transport between Membranes and NPC2†
- (2008) Zhi Xu et al. BIOCHEMISTRY
- Imatinib therapy blocks cerebellar apoptosis and improves neurological symptoms in a mouse model of Niemann-Pick type C disease
- (2008) Alejandra R. Alvarez et al. FASEB JOURNAL
- GM2/GD2 and GM3 gangliosides have no effect on cellular cholesterol pools or turnover in normal or NPC1 mice
- (2008) Hao Li et al. JOURNAL OF LIPID RESEARCH
- Niemann-Pick disease type C1 is a sphingosine storage disease that causes deregulation of lysosomal calcium
- (2008) Emyr Lloyd-Evans et al. NATURE MEDICINE
- Clinical, Electrophysiological, and Serum Biochemical Measures of Progressive Neurological and Hepatic Dysfunction in Feline Niemann-Pick Type C Disease
- (2008) Charles H Vite et al. PEDIATRIC RESEARCH
- NPC2 facilitates bidirectional transfer of cholesterol between NPC1 and lipid bilayers, a step in cholesterol egress from lysosomes
- (2008) R. E. Infante et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
Find Funding. Review Successful Grants.
Explore over 25,000 new funding opportunities and over 6,000,000 successful grants.
ExplorePublish scientific posters with Peeref
Peeref publishes scientific posters from all research disciplines. Our Diamond Open Access policy means free access to content and no publication fees for authors.
Learn More