Cyclodextrins applied to the treatment of lysosomal storage disorders

Inclusion Solves Insolubility —Translational Research Cycle from Bedside to Bench and Bench to Bedside for Drug Development Targeting Niemann-Pick Disease Type C—

(2022) Tetsumi Irie   YAKUGAKU ZASSHI-JOURNAL OF THE PHARMACEUTICAL SOCIETY OF JAPAN

Current treatment options and novel nanotechnology-driven enzyme replacement strategies for lysosomal storage disorders

(2022) Ambra Del Grosso et al.   ADVANCED DRUG DELIVERY REVIEWS

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

Gender-Specific Effects of Two Treatment Strategies in a Mouse Model of Niemann-Pick Disease Type C1

(2021) Carsten Holzmann et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Differential mode of cholesterol inclusion with 2‐hydroxypropyl‐cyclodextrins increases safety margin in treatment of Niemann‐Pick disease type C

(2021) Yusei Yamada et al.   BRITISH JOURNAL OF PHARMACOLOGY

Lipid-mediated motor-adaptor sequestration impairs axonal lysosome delivery leading to autophagic stress and dystrophy in Niemann-Pick type C

(2021) Joseph C. Roney et al.   DEVELOPMENTAL CELL

Fabry Disease: The Current Treatment Landscape

(2021) Malte Lenders et al.   DRUGS

One-year results of a clinical trial of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency

(2021) George A. Diaz et al.   GENETICS IN MEDICINE

Transcriptome of HPβCD-treated Niemann-pick disease type C1 cells highlights GPNMB as a biomarker for therapeutics

(2021) Jorge L Rodriguez-Gil et al.   HUMAN MOLECULAR GENETICS

Estimating the prevalence of Niemann-Pick disease type C (NPC) in the United States

(2021) Barbara K. Burton et al.   MOLECULAR GENETICS AND METABOLISM

Spatiotemporal Developmental Upregulation of Prestin Correlates With the Severity and Location of Cyclodextrin-Induced Outer Hair Cell Loss and Hearing Loss

(2021) Dalian Ding et al.   Frontiers in Cell and Developmental Biology

A human iPSC-derived inducible neuronal model of Niemann-Pick disease, type C1

(2021) Anika V. Prabhu et al.   BMC BIOLOGY

Acid Sphingomyelinase Deficiency: A Clinical and Immunological Perspective

(2021) Carolina Pinto et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial

(2021) Jordi Diaz-Manera et al.   LANCET NEUROLOGY

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

(2021) Amélie Barthelemy et al.   NEUROBIOLOGY OF DISEASE

Differential Effects of 2-Hydroxypropyl-Cyclodextrins on Lipid Accumulation in Npc1-Null Cells

(2020) Sanzana Hoque et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

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

Abnormal LAMP1 glycosylation may play a role in Niemann-Pick disease, type C pathology

(2020) Niamh X. Cawley et al.   PLoS One

Inherited disorders of lysosomal membrane transporters

(2020) Marjan Huizing et al.   BIOCHIMICA ET BIOPHYSICA ACTA-BIOMEMBRANES

2-Hydroxypropyl-gamma-cyclodextrin overcomes NPC1 deficiency by enhancing lysosome-ER association and autophagy

(2020) Ashutosh Singhal et al.   Scientific Reports

Identification of Brain-Specific Treatment Effects in NPC1 Disease by Focusing on Cellular and Molecular Changes of Sphingosine-1-Phosphate Metabolism

(2020) Anne Gläser et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Long-term efficacy of olipudase alfa in adults with acid sphingomyelinase deficiency (ASMD): Further clearance of hepatic sphingomyelin is associated with additional improvements in pro- and anti-atherogenic lipid profiles after 42 months of treatment

(2020) Beth L. Thurberg et al.   MOLECULAR GENETICS AND METABOLISM

2-Hydroxypropyl-β-cyclodextrin Ototoxicity in Adult Rats: Rapid Onset and Massive Destruction of Both Inner and Outer Hair Cells Above a Critical Dose

(2020) Xiaopeng Liu et al.   NEUROTOXICITY RESEARCH

Investigating the Mechanism of Cyclodextrins in the Treatment of Niemann‐Pick Disease Type C Using Crosslinked 2‐Hydroxypropyl‐β‐cyclodextrin

(2020) Dario Carradori et al.   Small

Lactose-appended β-cyclodextrin as an effective nanocarrier for brain delivery

(2020) Ryoma Yokoyama et al.   JOURNAL OF CONTROLLED RELEASE

Application of a glycinated bile acid biomarker for diagnosis and assessment of response to treatment in Niemann-pick disease type C1

(2020) Rohini Sidhu et al.   MOLECULAR GENETICS AND METABOLISM

Delineation of metabolic responses of Npc1 mice lacking the cholesterol-esterifying enzyme SOAT2 to acute treatment with 2-hydroxypropyl-β-cyclodextrin

(2020) Charina M. Ramirez et al.   STEROIDS

In Vitro and In Vivo Evaluation of 6-O-α-Maltosyl-β-Cyclodextrin as a Potential Therapeutic Agent Against Niemann-Pick Disease Type C

(2019) Nushrat Yasmin et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Treating Lysosomal Storage Disorders: What Have We Learnt?

(2019) Robin H Lachmann   JOURNAL OF INHERITED METABOLIC DISEASE

In vivo Efficacy and Safety Evaluation of Lactosyl-β-cyclodextrin as a Therapeutic Agent for Hepatomegaly in Niemann-Pick Type C Disease

(2019) Yuki Maeda et al.   Nanomaterials

Ontogenesis and Modulation of Intestinal Unesterified Cholesterol Sequestration in a Mouse Model of Niemann–Pick C1 Disease

(2019) Adam M. Lopez et al.   DIGESTIVE DISEASES AND SCIENCES

A therapy with miglustat, 2-hydroxypropyl-ß-cyclodextrin and allopregnanolone restores splenic cholesterol homeostasis in Niemann-pick disease type C1

(2019) Anna-Maria Neßlauer et al.   Lipids in Health and Disease

Expanded access with intravenous hydroxypropyl-β-cyclodextrin to treat children and young adults with Niemann-Pick disease type C1: a case report analysis

(2019) Caroline Hastings et al.   Orphanet Journal of Rare Diseases

Cyclodextrin polymer improves atherosclerosis therapy and reduces ototoxicity

(2019) Heegon Kim et al.   JOURNAL OF CONTROLLED RELEASE

Role of 6- O -α-maltosyl-β-cyclodextrin in lysosomal cholesterol deprivation in Npc1- deficient Chinese hamster ovary cells

(2018) Yasuyo Okada et al.   CARBOHYDRATE RESEARCH

In Niemann-Pick C1 mouse models, glial-only expression of the normal gene extends survival much further than do changes in genetic background or treatment with hydroxypropyl-beta-cyclodextrin

(2018) Craig A. Marshall et al.   GENE

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

(2018) Antony Cougnoux et al.   HUMAN MOLECULAR GENETICS

Evaluation of Two Liver Treatment Strategies in a Mouse Model of Niemann–Pick-Disease Type C1

(2018) Lynn Ebner et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

A pilot study of direct delivery of hydroxypropyl-beta-cyclodextrin to the lung by the nasal route in a mouse model of Niemann-Pick C1 disease: motor performance is unaltered and lung disease is worsened

(2018) Robert P. Erickson et al.   JOURNAL OF APPLIED GENETICS

Polyrotaxane-based systemic delivery of β-cyclodextrins for potentiating therapeutic efficacy in a mouse model of Niemann-Pick type C disease

(2018) Atsushi Tamura et al.   JOURNAL OF CONTROLLED RELEASE

LC3 Immunostaining in the Inferior Olivary Nuclei of Cats With Niemann-Pick Disease Type C1 Is Associated With Patterned Purkinje Cell Loss

(2018) Brittney L Gurda et al.   JOURNAL OF NEUROPATHOLOGY AND EXPERIMENTAL NEUROLOGY

Predicting the Binding Mode of 2-Hydroxypropyl-β-cyclodextrin to Cholesterol by Means of the MD Simulation and the 3D-RISM-KH Theory

(2018) Yuji Hayashino et al.   JOURNAL OF PHYSICAL CHEMISTRY B

Cyclodextrins: Assessing the Impact of Cavity Size, Occupancy, and Substitutions on Cytotoxicity and Cholesterol Homeostasis

(2018) Lajos Szente et al.   MOLECULES

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

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

Linear Cyclodextrin Polymer Prodrugs as Novel Therapeutics for Niemann-Pick Type C1 Disorder

(2018) Aditya Kulkarni et al.   Scientific Reports

The susceptibility of cochlear outer hair cells to cyclodextrin is not related to their electromotile activity

(2018) Yingjie Zhou et al.   Acta Neuropathologica Communications

Main Olfactory and Vomeronasal Epithelium Are Differently Affected in Niemann-Pick Disease Type C1

(2018) Martin Witt et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Necroptosis inhibition as a therapy for Niemann-Pick disease, type C1: Inhibition of RIP kinases and combination therapy with 2-hydroxypropyl-β-cyclodextrin

(2018) A. Cougnoux et al.   MOLECULAR GENETICS AND METABOLISM

Hydroxypropyl-beta and -gamma cyclodextrins rescue cholesterol accumulation in Niemann–Pick C1 mutant cell via lysosome-associated membrane protein 1

(2018) Ashutosh Singhal et al.   Cell Death & Disease

Shortened primary cilium length and dysregulated Sonic hedgehog signaling in Niemann-Pick C1 disease

(2017) Sonia Canterini et al.   HUMAN MOLECULAR GENETICS

Increased Regenerative Capacity of the Olfactory Epithelium in Niemann–Pick Disease Type C1

(2017) Anja Meyer et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Impact of the Niemann–Pick c1 Gene Mutation on the Total Cellular Glycomics of CHO Cells

(2017) Jun-ichi Furukawa et al.   JOURNAL OF PROTEOME RESEARCH

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

Early experience with compassionate use of 2 hydroxypropyl-beta-cyclodextrin for Niemann–Pick type C disease: review of initial published cases

(2017) Juan Eduardo Megías-Vericat et al.   NEUROLOGICAL SCIENCES

Neural stem cells for disease modeling of Wolman disease and evaluation of therapeutics

(2017) Francis Aguisanda et al.   Orphanet Journal of Rare Diseases

Cyclodextrins and Iatrogenic Hearing Loss: New Drugs with Significant Risk

(2017) Mark A. Crumling et al.   Frontiers in Cellular Neuroscience

Characterization of hydroxypropyl-beta-cyclodextrins used in the treatment of Niemann-Pick Disease type C1

(2017) Alfred L. Yergey et al.   PLoS One

Direct administration of 2-Hydroxypropyl-Beta-Cyclodextrin into guinea pig cochleae: Effects on physiological and histological measurements

(2017) J. T. Lichtenhan et al.   PLoS One

Noise-Induced Loss of Hair Cells and Cochlear Synaptopathy Are Mediated by the Activation of AMPK

(2016) K. Hill et al.   JOURNAL OF NEUROSCIENCE

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

In vitro evaluation of 2-hydroxyalkylated β-cyclodextrins as potential therapeutic agents for Niemann-Pick Type C disease

(2016) Yuki Kondo et al.   MOLECULAR GENETICS AND METABOLISM

Lysosomal pH-inducible supramolecular dissociation of polyrotaxanes possessing acid-labile N-triphenylmethyl end groups and their therapeutic potential for Niemann-Pick type C disease

(2016) Atsushi Tamura et al.   SCIENCE AND TECHNOLOGY OF ADVANCED MATERIALS

PEG-lipid micelles enable cholesterol efflux in Niemann-Pick Type C1 disease-based lysosomal storage disorder

(2016) Anna Brown et al.   Scientific Reports

Induced Pluripotent Stem Cells for Disease Modeling and Evaluation of Therapeutics for Niemann-Pick Disease Type A

(2016) Yan Long et al.   Stem Cells Translational Medicine

Efficacy and ototoxicity of different cyclodextrins in Niemann-Pick C disease

(2016) Cristin D. Davidson et al.   Annals of Clinical and Translational Neurology

Developmental delay in motor skill acquisition in Niemann-Pick C1 mice reveals abnormal cerebellar morphogenesis

(2016) Paola Caporali et al.   Acta Neuropathologica Communications

Susceptibility of outer hair cells to cholesterol chelator 2-hydroxypropyl-β-cyclodextrine is prestin-dependent

(2016) Satoe Takahashi et al.   Scientific Reports

Cholesterol lowering effects of mono-lactose-appended β-cyclodextrin in Niemann–Pick type C disease-like HepG2 cells

(2015) Keiichi Motoyama et al.   Beilstein Journal of Organic Chemistry

Efficacy of 2-Hydroxypropyl-β-cyclodextrin in Niemann–Pick Disease Type C Model Mice and Its Pharmacokinetic Analysis in a Patient with the Disease

(2015) Yuta Tanaka et al.   BIOLOGICAL & PHARMACEUTICAL BULLETIN

Immunogenicity of Elosulfase Alfa, an Enzyme Replacement Therapy in Patients With Morquio A Syndrome: Results From MOR-004, a Phase III Trial

(2015) Becky Schweighardt et al.   CLINICAL THERAPEUTICS

Enhanced delivery of daidzein into fibroblasts and neuronal cells with cationic derivatives of gamma-cyclodextrin for the control of cellular glycosaminoglycans

(2015) Kamil Kamiński et al.   EUROPEAN JOURNAL OF PHARMACEUTICS AND BIOPHARMACEUTICS

BCM-95 and (2-hydroxypropyl)-β-cyclodextrin reverse autophagy dysfunction and deplete stored lipids in Sap C-deficient fibroblasts

(2015) Massimo Tatti et al.   HUMAN MOLECULAR GENETICS

Hearing Loss and Otopathology Following Systemic and Intracerebroventricular Delivery of 2-Hydroxypropyl-Beta-Cyclodextrin

(2015) Scott Cronin et al.   JARO-JOURNAL OF THE ASSOCIATION FOR RESEARCH IN OTOLARYNGOLOGY

β-Cyclodextrin-threaded Biocleavable Polyrotaxanes Ameliorate Impaired Autophagic Flux in Niemann-Pick Type C Disease

(2015) Atsushi Tamura et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Effects of cyclodextrins on GM1-gangliosides in fibroblasts from GM1-gangliosidosis patients

(2015) Yuki Maeda et al.   JOURNAL OF PHARMACY AND PHARMACOLOGY

Intrathecal 2-Hydroxypropyl-Beta-Cyclodextrin (HPBCD) therapy in adult-onset Niemann-Pick Disease Type C (NPC)

(2015) Y. Sakiyama et al.   JOURNAL OF THE NEUROLOGICAL SCIENCES

Intrathecal 2-hydroxypropyl-beta-cyclodextrin in a single patient with Niemann–Pick C1

(2015) Timothy J. Maarup et al.   MOLECULAR GENETICS AND METABOLISM

How to reduce the accumulation of autophagic vacuoles in NPC1-deficient neurons: A comparison of two pharmacological strategies

(2015) Volker Meske et al.   NEUROPHARMACOLOGY

A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase Deficiency

(2015) Barbara K. Burton et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

HPGCD Outperforms HPBCD as a Potential Treatment for Niemann-Pick Disease Type C During Disease Modeling with iPS Cells

(2015) Minami Soga et al.   STEM CELLS

Cyclodextrins, Blood–Brain Barrier, and Treatment of Neurological Diseases

(2014) Miklós Vecsernyés et al.   ARCHIVES OF MEDICAL RESEARCH

Plasma Signature of Neurological Disease in the Monogenetic Disorder Niemann-Pick Type C

(2014) Md. Suhail Alam et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Complex lipid trafficking in Niemann-Pick disease type C

(2014) Marie T. Vanier   JOURNAL OF INHERITED METABOLIC DISEASE

Reduced cerebellar neurodegeneration after combined therapy with cyclodextrin/allopregnanolone and miglustat in NPC1: A mouse model of Niemann-Pick type C1 disease

(2014) Fabian Maass et al.   JOURNAL OF NEUROSCIENCE RESEARCH

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

A marked paucity of granule cells in the developing cerebellum of the Npc1−/− mouse is corrected by a single injection of hydroxypropyl-β-cyclodextrin

(2014) S. Nusca et al.   NEUROBIOLOGY OF DISEASE

Targeted delivery of pharmacological chaperones for Gaucher disease to macrophages by a mannosylated cyclodextrin carrier

(2014) Julio Rodríguez-Lavado et al.   ORGANIC & BIOMOLECULAR CHEMISTRY

Lysosomal-specific Cholesterol Reduction by Biocleavable Polyrotaxanes for Ameliorating Niemann-Pick Type C Disease

(2014) Atsushi Tamura et al.   Scientific Reports

Genetic and Chemical Correction of Cholesterol Accumulation and Impaired Autophagy in Hepatic and Neural Cells Derived from Niemann-Pick Type C Patient-Specific iPS Cells

(2014) Dorothea Maetzel et al.   Stem Cell Reports

Auditory Phenotype of Niemann-Pick Disease, Type C1

(2013) Kelly A. King et al.   EAR AND HEARING

Galsulfase (Naglazyme®) therapy in infants with mucopolysaccharidosis VI

(2013) Paul R. Harmatz et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Identification of Niemann-Pick C1 disease biomarkers through sphingolipid profiling

(2013) Martin Fan et al.   JOURNAL OF LIPID RESEARCH

Gangliosides and Gangliosidoses: Principles of Molecular and Metabolic Pathogenesis

(2013) K. Sandhoff et al.   JOURNAL OF NEUROSCIENCE

Lung toxicity of hydroxypropyl-β-cyclodextrin infusion

(2013) Yin-Hsiu Chien et al.   MOLECULAR GENETICS AND METABOLISM

Combined therapy with cyclodextrin/allopregnanolone and miglustat improves motor but not cognitive functions in Niemann–Pick Type C1 mice

(2013) M. Hovakimyan et al.   NEUROSCIENCE

Impaired Autophagy in the Lipid-Storage Disorder Niemann-Pick Type C1 Disease

(2013) Sovan Sarkar et al.   Cell Reports

Microarray expression analysis and identification of serum biomarkers for Niemann–Pick disease, type C1

(2012) Celine V.M. Cluzeau et al.   HUMAN MOLECULAR GENETICS

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

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

Cyclodextrin mediates rapid changes in lipid balance inNpc1−/−mice without carrying cholesterol through the bloodstream

(2012) Anna M. Taylor et al.   JOURNAL OF LIPID RESEARCH

Amyloid-β metabolism in Niemann-Pick C disease models and patients

(2012) Niklas Mattsson et al.   METABOLIC BRAIN DISEASE

Recommendations for the diagnosis and management of Niemann–Pick disease type C: An update

(2012) Marc C. Patterson et al.   MOLECULAR GENETICS AND METABOLISM

Effects of cyclodextrin in two patients with Niemann–Pick Type C disease

(2012) Muneaki Matsuo et al.   MOLECULAR GENETICS AND METABOLISM

Hearing Loss and Hair Cell Death in Mice Given the Cholesterol-Chelating Agent Hydroxypropyl-β-Cyclodextrin

(2012) Mark A. Crumling et al.   PLoS One

Long-Term Parenteral Administration of 2-Hydroxypropyl-β-Cyclodextrin Causes Bone Loss

(2012) Ingrid Kantner et al.   TOXICOLOGIC PATHOLOGY

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

Corneal Alterations during Combined Therapy with Cyclodextrin/Allopregnanolone and Miglustat in a Knock-Out Mouse Model of NPC1 Disease

(2011) Marine Hovakimyan et al.   PLoS One

Molecular Mechanism of Cyclodextrin Mediated Cholesterol Extraction

(2011) Cesar A. López et al.   PLoS Computational Biology

Protection afforded by a herbal medicine, Sho-seiryu-to (TJ-19), against oleic acid-induced acute lung injury in guinea-pigs

(2010) Chang-qing Yang et al.   JOURNAL OF PHARMACY AND PHARMACOLOGY

Pharmaceutical applications of cyclodextrins: basic science and product development

(2010) Thorsteinn Loftsson et al.   JOURNAL OF PHARMACY AND PHARMACOLOGY

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

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

Effects of Bolus Injection of Soybean-Based Fat Emulsion and Fatty Acids on Pulmonary Gas Exchange Function

(2009) Yoichi Ishitsuka et al.   BIOLOGICAL & PHARMACEUTICAL BULLETIN

Structure of N-Terminal Domain of NPC1 Reveals Distinct Subdomains for Binding and Transfer of Cholesterol

(2009) Hyock Joo Kwon et al.   CELL

A Selective Thromboxane A2 (TXA2) Synthase Inhibitor, Ozagrel, Attenuates Lung Injury and Decreases Monocyte Chemoattractant Protein-1 and Interleukin-8 mRNA Expression in Oleic Acid–Induced Lung Injury in Guinea Pigs

(2009) Yoichi Ishitsuka et al.   JOURNAL OF PHARMACOLOGICAL SCIENCES

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

The pathogenesis of Niemann–Pick type C disease: a role for autophagy?

(2008) Chris D. Pacheco et al.   EXPERT REVIEWS IN MOLECULAR MEDICINE

Study of the cholesterol extraction capacity of β-cyclodextrin and its derivatives, relationships with their effects on endothelial cell viability and on membrane models

(2008) Delphine Castagne et al.   JOURNAL OF INCLUSION PHENOMENA AND MACROCYCLIC CHEMISTRY