Glial contribution to cyclodextrin-mediated reversal of cholesterol accumulation in murine NPC1-deficient neurons in vivo

Intracerebroventricular Treatment with 2-Hydroxypropyl-β-Cyclodextrin Decreased Cerebellar and Hepatic Glycoprotein Nonmetastatic Melanoma Protein B (GPNMB) Expression in Niemann–Pick Disease Type C Model Mice

(2021) Madoka Fukaura et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Loss of NPC1 enhances phagocytic uptake and impairs lipid trafficking in microglia

(2021) Alessio Colombo et al.   Nature Communications

Genistein Activates Transcription Factor EB and Corrects Niemann–Pick C Phenotype

(2021) Graciela Argüello et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Microglia reprogram metabolic profiles for phenotype and function changes in central nervous system

(2021) Sheng Yang et al.   NEUROBIOLOGY OF DISEASE

Nanoparticles containing β‐cyclodextrin potentially useful for the treatment of Niemann‐Pick C

(2020) Bruna Donida et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Monitoring the itinerary of lysosomal cholesterol in Niemann-Pick Type C1-deficient cells after cyclodextrin treatment

(2020) McKenna Feltes et al.   JOURNAL OF LIPID RESEARCH

Mechanism of Secondary Ganglioside and Lipid Accumulation in Lysosomal Disease

(2020) Bernadette Breiden et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Retinal axonal degeneration in Niemann–Pick type C disease

(2020) Joachim Havla et al.   JOURNAL OF NEUROLOGY

Imaging of neuroinflammation in adult Niemann-Pick type C disease

(2020) Mark Walterfang et al.   NEUROLOGY

Lipid and Lipoprotein Metabolism in Microglia

(2020) Bailey A. Loving et al.   Frontiers in Physiology

Microglial Corpse Clearance: Lessons From Macrophages

(2020) Mar Márquez-Ropero et al.   Frontiers in Immunology

Structural Basis of Low-pH-Dependent Lysosomal Cholesterol Egress by NPC1 and NPC2

(2020) Hongwu Qian et al.   CELL

Single Cell Transcriptome Analysis of Niemann–Pick Disease, Type C1 Cerebella

(2020) Antony Cougnoux et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Microglia and lipids: how metabolism controls brain innate immunity

(2020) Bruno Chausse et al.   SEMINARS IN CELL & DEVELOPMENTAL BIOLOGY

Understanding and Treating Niemann–Pick Type C Disease: Models Matter

(2020) Valentina Pallottini et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Deregulation of signalling in genetic conditions affecting the lysosomal metabolism of cholesterol and galactosyl-sphingolipids

(2020) S. Gowrishankar et al.   NEUROBIOLOGY OF DISEASE

Cyclodextrin triggers MCOLN1-dependent endo-lysosome secretion in Niemann-Pick type C cells

(2019) Fabrizio Vacca et al.   JOURNAL OF LIPID RESEARCH

NPC intracellular cholesterol transporter 1 (NPC1)-mediated cholesterol export from lysosomes

(2019) Suzanne R. Pfeffer   JOURNAL OF BIOLOGICAL CHEMISTRY

Glial phagocytic clearance in Parkinson’s disease

(2019) Marie-Eve Tremblay et al.   Molecular Neurodegeneration

Characterization of the Subventricular-Thalamo-Cortical Circuit in the NP-C Mouse Brain, and New Insights Regarding Treatment

(2019) Min Hee Park et al.   MOLECULAR THERAPY

Long-Term Neuropsychological Outcomes from an Open-Label Phase I/IIa Trial of 2-Hydroxypropyl-β-Cyclodextrins (VTS-270) in Niemann-Pick Disease, Type C1

(2019) Cristan A. Farmer et al.   CNS DRUGS

Modest Blood-brain Barrier Permeability of the Cyclodextrin Kleptose®: Modification by Efflux and Luminal Surface Binding

(2019) William A. Banks et al.   JOURNAL OF PHARMACOLOGY AND EXPERIMENTAL THERAPEUTICS

Phagocytosis in the Brain: Homeostasis and Disease

(2019) Dylan A. Galloway et al.   Frontiers in Immunology

The complexity of a monogenic neurodegenerative disease: More than two decades of therapeutic driven research into Niemann-Pick type C disease

(2019) Natalie Hammond et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR AND CELL BIOLOGY OF LIPIDS

Current Challenges in Understanding the Cellular and Molecular Mechanisms in Niemann–Pick Disease Type C1

(2019) Anja U. Bräuer et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Intravitreal Pharmacokinetics in Mice: SPECT/CT Imaging and Scaling to Rabbits and Humans

(2019) Mechthild Schmitt et al.   MOLECULAR PHARMACEUTICS

Neutrophil Extracellular Traps in Tissue and Periphery in Juvenile Dermatomyositis

(2019) Bhargavi Duvvuri et al.   Arthritis & Rheumatology

Pharmacokinetic Properties of Fluorescently Labelled Hydroxypropyl-Beta-Cyclodextrin

(2019) Váradi et al.   Biomolecules

Microglia Biology: One Century of Evolving Concepts

(2019) Marco Prinz et al.   CELL

Structural Insight into Eukaryotic Sterol Transport through Niemann-Pick Type C Proteins

(2019) Mikael B.L. Winkler et al.   CELL

Niemann–Pick type C disease: cellular pathology and pharmacotherapy

(2019) Simon Wheeler et al.   JOURNAL OF NEUROCHEMISTRY

Addressing neurodegeneration in lysosomal storage disorders: Advances in Niemann Pick diseases

(2019) Ana Toledano-Zaragoza et al.   NEUROPHARMACOLOGY

Lysosomal integral membrane protein-2 (LIMP-2/SCARB2) is involved in lysosomal cholesterol export

(2019) Saskia Heybrock et al.   Nature Communications

Increased interactions and engulfment of dendrites by microglia precede Purkinje cell degeneration in a mouse model of Niemann Pick Type-C

(2019) Larisa Kavetsky et al.   Scientific Reports

Microglia activation in Niemann–Pick disease, type C1 is amendable to therapeutic intervention

(2018) Antony Cougnoux et al.   HUMAN MOLECULAR GENETICS

Gold nanoparticles in ophthalmology

(2018) Florence Masse et al.   MEDICINAL RESEARCH REVIEWS

Consensus clinical management guidelines for Niemann-Pick disease type C

(2018) Tarekegn Geberhiwot et al.   Orphanet Journal of Rare Diseases

Long-Term Treatment of Niemann-Pick Type C1 Disease With Intrathecal 2-Hydroxypropyl-β-Cyclodextrin

(2018) Elizabeth Berry-Kravis et al.   PEDIATRIC NEUROLOGY

Lipid‐induced lysosomal damage after demyelination corrupts microglia protective function in lysosomal storage disorders

(2018) Enrique Gabandé‐Rodríguez et al.   EMBO JOURNAL

Retinal Vascular Abnormalities and Microglia Activation in Mice with Deficiency in Cytochrome P450 46A1–Mediated Cholesterol Removal

(2018) Aicha Saadane et al.   AMERICAN JOURNAL OF PATHOLOGY

Cyclodextrin-Modified inorganic materials for the construction of nanocarriers

(2017) Giovanna Cutrone et al.   INTERNATIONAL JOURNAL OF PHARMACEUTICS

Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1–2 trial

(2017) Daniel S Ory et al.   LANCET

3.3 Å structure of Niemann–Pick C1 protein reveals insights into the function of the C-terminal luminal domain in cholesterol transport

(2017) Xiaochun Li et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Pharmacokinetic aspects of retinal drug delivery

(2017) Eva M. del Amo et al.   PROGRESS IN RETINAL AND EYE RESEARCH

Dark microglia: A new phenotype predominantly associated with pathological states

(2016) Kanchan Bisht et al.   GLIA

Reversal of Pathologic Lipid Accumulation in NPC1-Deficient Neurons by Drug-Promoted Release of LAMP1-Coated Lamellar Inclusions

(2016) Valérie Demais et al.   JOURNAL OF NEUROSCIENCE

Cyclodextrins as Emerging Therapeutic Tools in the Treatment of Cholesterol-Associated Vascular and Neurodegenerative Diseases

(2016) Caroline Coisne et al.   MOLECULES

Glia–neuron interactions in the mammalian retina

(2016) Elena Vecino et al.   PROGRESS IN RETINAL AND EYE RESEARCH

A Murine Niemann-Pick C1 I1061T Knock-In Model Recapitulates the Pathological Features of the Most Prevalent Human Disease Allele

(2015) M. Praggastis et al.   JOURNAL OF NEUROSCIENCE

Visual evoked potentials of Niemann-Pick type C1 mice reveal an impairment of the visual pathway that is rescued by 2-hydroxypropyl-ß-cyclodextrin

(2015) Giampiero Palladino et al.   Orphanet Journal of Rare Diseases

Intracisternal cyclodextrin prevents cerebellar dysfunction and Purkinje cell death in feline Niemann-Pick type C1 disease

(2015) Charles H. Vite et al.   Science Translational Medicine

Identification of NPC1 as the target of U18666A, an inhibitor of lysosomal cholesterol export and Ebola infection

(2015) Feiran Lu et al.   eLife

Excessive microglial activation aggravates olfactory dysfunction by impeding the survival of newborn neurons in the olfactory bulb of Niemann–Pick disease type C1 mice

(2014) Yoojin Seo et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE

Defects in the retina of Niemann-pick type C 1 mutant mice

(2014) Xin Yan et al.   BMC NEUROSCIENCE

Review: A History of Cyclodextrins

(2014) Grégorio Crini   CHEMICAL REVIEWS

Hyperactive glial cells contribute to axonal pathologies in the spinal cord of Npc1 mutant mice

(2014) Xin Yan et al.   GLIA

Niemann-Pick C disease and mobilization of lysosomal cholesterol by cyclodextrin

(2014) Jean E. Vance et al.   JOURNAL OF LIPID RESEARCH

Relevance of neuronal and glial NPC1 for synaptic input to cerebellar Purkinje cells

(2014) Isabelle Buard et al.   MOLECULAR AND CELLULAR NEUROSCIENCE

The autophagic defect in Niemann–Pick disease type C neurons differs from somatic cells and reduces neuronal viability

(2014) Volker Meske et al.   NEUROBIOLOGY OF DISEASE

Visualization of cholesterol deposits in lysosomes of Niemann-Pick type C fibroblasts using recombinant perfringolysin O

(2014) Katarzyna Kwiatkowska et al.   Orphanet Journal of Rare Diseases

Cholesterol in the retina: The best is yet to come

(2014) Irina A. Pikuleva et al.   PROGRESS IN RETINAL AND EYE RESEARCH

Cyclodextrin alleviates neuronal storage of cholesterol in Niemann-Pick C disease without evidence of detectable blood–brain barrier permeability

(2013) Charles C. Pontikis et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Human and mouse neuroinflammation markers in Niemann-Pick disease, type C1

(2013) Stephanie M. Cologna et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Simvastatin Treatment Highlights a New Role for the Isoprenoid/Cholesterol Biosynthetic Pathway in the Modulation of Emotional Reactivity and Cognitive Performance in Rats

(2013) Marco Segatto et al.   NEUROPSYCHOPHARMACOLOGY

Uptake of a fluorescent methyl-β-cyclodextrin via clathrin-dependent endocytosis

(2012) Anna Pia Plazzo et al.   CHEMISTRY AND PHYSICS OF LIPIDS

Mutant human APP exacerbates pathology in a mouse model of NPC and its reversal by a β-cyclodextrin

(2012) Mahua Maulik et al.   HUMAN MOLECULAR GENETICS

Cyclodextrins

(2012) Sergey V. Kurkov et al.   INTERNATIONAL JOURNAL OF PHARMACEUTICS

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

Miglustat Improves Purkinje Cell Survival and Alters Microglial Phenotype in Feline Niemann-Pick Disease Type C

(2012) Veronika M. Stein et al.   JOURNAL OF NEUROPATHOLOGY AND EXPERIMENTAL NEUROLOGY

Infection-induced NETosis is a dynamic process involving neutrophil multitasking in vivo

(2012) Bryan G Yipp et al.   NATURE MEDICINE

Niemann-Pick Type C1 deficiency in microglia does not cause neuron death in vitro

(2011) Kyle B. Peake et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE

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

Anatomically Defined Neuron-Based Rescue of Neurodegenerative Niemann-Pick Type C Disorder

(2011) M. E. Lopez et al.   JOURNAL OF NEUROSCIENCE

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

Early glial activation, synaptic changes and axonal pathology in the thalamocortical system of Niemann–Pick type C1 mice

(2011) Sarah N.R. Pressey et al.   NEUROBIOLOGY OF DISEASE

Cholesterol metabolism in neurons and astrocytes

(2011) Frank W. Pfrieger et al.   PROGRESS IN LIPID RESEARCH

Niemann-Pick disease type C

(2010) Marie T Vanier   Orphanet Journal of Rare Diseases

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

Cyclodextrin Induces Calcium-Dependent Lysosomal Exocytosis

(2010) Fannie W. Chen et al.   PLoS One

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

Marked differences in cholesterol synthesis between neurons and glial cells from postnatal rats

(2009) Katja Nieweg et al.   JOURNAL OF NEUROCHEMISTRY

Lack of Niemann–Pick type C1 induces age-related degeneration in the mouse retina

(2009) Thomas Claudepierre et al.   MOLECULAR AND CELLULAR NEUROSCIENCE

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

Neuronal Loss of Drosophila NPC1a Causes Cholesterol Aggregation and Age-Progressive Neurodegeneration

(2008) S. E. Phillips et al.   JOURNAL OF NEUROSCIENCE

NS21: Re-defined and modified supplement B27 for neuronal cultures

(2008) Yucui Chen et al.   JOURNAL OF NEUROSCIENCE METHODS

Brainstem neuropathology in a mouse model of Niemann–Pick disease type C

(2008) Zhuo Luan et al.   JOURNAL OF THE NEUROLOGICAL SCIENCES

Niemann-Pick disease type C1 is a sphingosine storage disease that causes deregulation of lysosomal calcium

(2008) Emyr Lloyd-Evans et al.   NATURE MEDICINE