Collaborative development of 2-hydroxypropyl-β-cyclodextrin for the treatment of Niemann-Pick type C1 disease
about
Susceptibility of outer hair cells to cholesterol chelator 2-hydroxypropyl-β-cyclodextrine is prestin-dependentRecent advances in the diagnosis and treatment of niemann-pick disease type C in children: a guide to early diagnosis for the general pediatricianDisruption of Lipid Raft Function Increases Expression and Secretion of Monocyte Chemoattractant Protein-1 in 3T3-L1 AdipocytesDefective Cytochrome P450-Catalysed Drug Metabolism in Niemann-Pick Type C DiseaseFostering collaborative research for rare genetic disease: the example of niemann-pick type C diseaseVisual evoked potentials of Niemann-Pick type C1 mice reveal an impairment of the visual pathway that is rescued by 2-hydroxypropyl-ß-cyclodextrinIntracisternal cyclodextrin prevents cerebellar dysfunction and Purkinje cell death in feline Niemann-Pick type C1 disease.Development and validation of sensitive LC-MS/MS assays for quantification of HP-β-CD in human plasma and CSF.Analytical Characterization of Methyl-β-Cyclodextrin for Pharmacological Activity to Reduce Lysosomal Cholesterol Accumulation in Niemann-Pick Disease Type C1 Cells.A novel mouse model of a patient mucolipidosis II mutation recapitulates disease pathologyCholesterol homeostatic responses provide biomarkers for monitoring treatment for the neurodegenerative disease Niemann-Pick C1 (NPC1)Cholesterol in brain disease: sometimes determinant and frequently implicated.High incidence of unrecognized visceral/neurological late-onset Niemann-Pick disease, type C1, predicted by analysis of massively parallel sequencing data setsA validated LC-MS/MS assay for quantification of 24(S)-hydroxycholesterol in plasma and cerebrospinal fluid.A novel, highly sensitive and specific biomarker for Niemann-Pick type C1 disease.Intrathecal 2-hydroxypropyl-beta-cyclodextrin in a single patient with Niemann-Pick C1.Identification of novel bile acids as biomarkers for the early diagnosis of Niemann-Pick C disease.Intravenous Formulation of HET0016 Decreased Human Glioblastoma Growth and Implicated Survival Benefit in Rat Xenograft ModelsDisease models for the development of therapies for lysosomal storage diseases.Cyclodextrins: improving the therapeutic response of analgesic drugs: a patent review.Modeling Niemann Pick type C1 using human embryonic and induced pluripotent stem cells.Advantages of Structure-Based Drug design Approaches in Neurological Disorders.Cyclodextrins as Protective Agents of Protein Aggregation: An Overview.Targeting cholesterol with β-cyclodextrin sensitizes cancer cells for apoptosis.Early experience with compassionate use of 2 hydroxypropyl-beta-cyclodextrin for Niemann-Pick type C disease: review of initial published cases.Cellular Cholesterol Homeostasis in Alzheimer's Disease.Reduction of TMEM97 increases NPC1 protein levels and restores cholesterol trafficking in Niemann-pick type C1 disease cells.NPC1 is enriched in unexplained early onset ataxia: a targeted high-throughput screening.PEG-lipid micelles enable cholesterol efflux in Niemann-Pick Type C1 disease-based lysosomal storage disorder.Hearing Loss and Otopathology Following Systemic and Intracerebroventricular Delivery of 2-Hydroxypropyl-Beta-Cyclodextrin.Precision Medicine in Cats: Novel Niemann-Pick Type C1 Diagnosed by Whole-Genome SequencingEffects of intracerebroventricular administration of 2-hydroxypropyl-β-cyclodextrin in a patient with Niemann-Pick Type C diseaseFTY720/fingolimod increases NPC1 and NPC2 expression and reduces cholesterol and sphingolipid accumulation in Niemann-Pick type C mutant fibroblasts.Quantitative Analysis of the Proteome Response to the Histone Deacetylase Inhibitor (HDACi) Vorinostat in Niemann-Pick Type C1 disease.Cyclodextrins and Iatrogenic Hearing Loss: New Drugs with Significant Risk.2-hydroxypropyl-β-cyclodextrins and the Blood-Brain Barrier: Considerations for Niemann-Pick Disease Type C1.Cyclodextrins improving the physicochemical and pharmacological properties of antidepressant drugs: a patent review.Is 2-Hydroxypropyl-β-cyclodextrin a Suitable Carrier for Central Administration of Δ9 -Tetrahydrocannabinol? Preclinical Evidence.Cyclodextrin has conflicting actions on autophagy flux in vivo in brains of normal and Alzheimer model mice.Hydroxypropyl-β-CD vs. its α-homologue for a 3D modified polyrotaxane network formation and properties: the relationship between modified CD and polymer revealed through comparison.
P2860
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P2860
Collaborative development of 2-hydroxypropyl-β-cyclodextrin for the treatment of Niemann-Pick type C1 disease
description
2014 nî lūn-bûn
@nan
2014 թուականին հրատարակուած գիտական յօդուած
@hyw
2014 թվականին հրատարակված գիտական հոդված
@hy
2014年の論文
@ja
2014年論文
@yue
2014年論文
@zh-hant
2014年論文
@zh-hk
2014年論文
@zh-mo
2014年論文
@zh-tw
2014年论文
@wuu
name
Collaborative development of 2 ...... f Niemann-Pick type C1 disease
@ast
Collaborative development of 2 ...... f Niemann-Pick type C1 disease
@en
Collaborative development of 2 ...... f Niemann-Pick type C1 disease
@nl
type
label
Collaborative development of 2 ...... f Niemann-Pick type C1 disease
@ast
Collaborative development of 2 ...... f Niemann-Pick type C1 disease
@en
Collaborative development of 2 ...... f Niemann-Pick type C1 disease
@nl
prefLabel
Collaborative development of 2 ...... f Niemann-Pick type C1 disease
@ast
Collaborative development of 2 ...... f Niemann-Pick type C1 disease
@en
Collaborative development of 2 ...... f Niemann-Pick type C1 disease
@nl
P2093
P2860
P50
P3181
P1476
Collaborative development of 2 ...... f Niemann-Pick type C1 disease
@en
P2093
Alan Hubbs
Charles H Vite
Daniel S Ory
Elizabeth A Ottinger
Forbes D Porter
Ilona Scott
Jim Cradock
John C McKew
Juan Marugan
Lili Portilla
P2860
P304
P3181
P356
10.2174/1568026613666131127160118
P407
P577
2014-01-01T00:00:00Z