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

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

Noninvasive hippocampal blood−brain barrier opening in Alzheimer’s disease with focused ultrasound

(2020) Ali R. Rezai et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Transcranial focused ultrasound, pulsed at 40 Hz, activates microglia acutely and reduces Aβ load chronically, as demonstrated in vivo

(2020) M.S. Bobola et al.   Brain Stimulation

Disease characteristics, prognosis and miglustat treatment effects on disease progression in patients with Niemann-Pick disease Type C: an international, multicenter, retrospective chart review

(2019) Mercedes Pineda et al.   Orphanet Journal of Rare Diseases

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

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

Animal models for Niemann-Pick type C: implications for drug discovery & development

(2019) Cathrine K. Fog et al.   Expert Opinion on Drug Discovery

Cholesterol depletion by MβCD enhances cell membrane tension, its variations and reduces integrity

(2019) Arikta Biswas et al.   BIOPHYSICAL JOURNAL

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

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

Adenosine A2A receptor stimulation restores cell functions and differentiation in Niemann-Pick type C-like oligodendrocytes

(2019) Chiara De Nuccio et al.   Scientific Reports

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

The Palliative Use of Intrathecal Baclofen in Niemann-Pick Disease Type C

(2019) Ana Ubeda Tikkanen et al.   PEDIATRICS

Focused Ultrasound Preconditioning for Augmented Nanoparticle Penetration and Efficacy in the Central Nervous System

(2019) Brian P. Mead et al.   Small

2-Hydroxypropyl-β-cyclodextrins and the Blood-Brain Barrier: Considerations for Niemann-Pick Disease Type C1

(2018) Pericles Calias   CURRENT PHARMACEUTICAL DESIGN

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

Safe and stable noninvasive focal gene delivery to the mammalian brain following focused ultrasound

(2018) Mihaela A. Stavarache et al.   JOURNAL OF NEUROSURGERY

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

(2018) Lajos Szente et al.   MOLECULES

Blood–brain barrier opening in Alzheimer’s disease using MR-guided focused ultrasound

(2018) Nir Lipsman et al.   Nature Communications

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

(2018) Aditya Kulkarni et al.   Scientific Reports

A Review of the Current Therapies, Challenges, and Future Directions of Transcranial Focused Ultrasound Technology

(2018) Vibhor Krishna et al.   JAMA Neurology

Cellular Uptake of Nanoparticles versus Small Molecules: A Matter of Size

(2018) Jesús Mosquera et al.   ACCOUNTS OF CHEMICAL RESEARCH

Cyclodextrin-Based Macromolecular Systems as Cholesterol-Mopping Therapeutic Agents in Niemann-Pick Disease Type C

(2018) Antonino Puglisi et al.   MACROMOLECULAR RAPID COMMUNICATIONS

The origin of neural stem cells impacts their interactions with targeted-lipid nanocapsules: Potential role of plasma membrane lipid composition and fluidity

(2018) Dario Carradori et al.   JOURNAL OF CONTROLLED RELEASE

Closed-loop cavitation control for focused ultrasound-mediated blood-brain-barrier opening by long-circulating microbubbles

(2018) Mustafa Cavusoglu et al.   PHYSICS IN MEDICINE AND BIOLOGY

Chemotherapy sensitization of glioblastoma by focused ultrasound-mediated delivery of therapeutic liposomes

(2018) Alexandros Papachristodoulou et al.   JOURNAL OF CONTROLLED RELEASE

Radiochemical synthesis and preclinical evaluation of 68Ga-labeled NODAGA-hydroxypropyl-beta-cyclodextrin (68Ga-NODAGA-HPBCD)

(2018) István Hajdu et al.   EUROPEAN JOURNAL OF PHARMACEUTICAL SCIENCES

FTY720/fingolimod increases NPC1 and NPC2 expression and reduces cholesterol and sphingolipid accumulation in Niemann-Pick type C mutant fibroblasts

(2017) Jason Newton et al.   FASEB JOURNAL

Niemann-Pick disease, type C and Roscoe Brady

(2017) Marc C. Patterson et al.   MOLECULAR GENETICS AND METABOLISM

Ror2 signaling regulates Golgi structure and transport through IFT20 for tumor invasiveness

(2017) Michiru Nishita et al.   Scientific Reports

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

(2016) Caroline Coisne et al.   MOLECULES

Heat shock protein–based therapy as a potential candidate for treating the sphingolipidoses

(2016) Thomas Kirkegaard et al.   Science Translational Medicine

In Vitro and In Vivo Evaluation of PEGylated Layer-by-Layer Polyelectrolyte-Coated Paclitaxel Nanocrystals

(2016) Anna Polomska et al.   Small

Rapid quantification of 2-hydroxypropyl-β-cyclodextrin in liquid pharmaceutical formulations by 1H nuclear magnetic resonance spectroscopy

(2015) Gilles Dufour et al.   EUROPEAN JOURNAL OF PHARMACEUTICAL SCIENCES

Pharmacokinetics of cyclodextrins and drugs after oral and parenteral administration of drug/cyclodextrin complexes

(2015) Thorsteinn Loftsson et al.   JOURNAL OF PHARMACY AND PHARMACOLOGY

Long term follow-up to evaluate the efficacy of miglustat treatment in Italian patients with Niemann-Pick disease type C

(2015) Simona Fecarotta et al.   Orphanet Journal of Rare Diseases

Rhodamine-labeled cyclodextrin derivatives

(2015) M. Malanga et al.   JOURNAL OF DRUG DELIVERY SCIENCE AND TECHNOLOGY

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

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

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

Alzheimer Disease in a Mouse Model: MR Imaging–guided Focused Ultrasound Targeted to the Hippocampus Opens the Blood-Brain Barrier and Improves Pathologic Abnormalities and Behavior

(2014) Alison Burgess et al.   RADIOLOGY

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

(2014) Atsushi Tamura et al.   Scientific Reports

Noninvasive and Targeted Drug Delivery to the Brain Using Focused Ultrasound

(2013) Alison Burgess et al.   ACS Chemical Neuroscience

Aspects of determining the molecular weight of cyclodextrin polymers and oligomers by static light scattering

(2013) István Puskás et al.   CARBOHYDRATE POLYMERS

Poly-(cyclo)dextrins as ethoxzolamide carriers in ophthalmic solutions and in contact lenses

(2013) M.J. García-Fernández et al.   CARBOHYDRATE POLYMERS

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

Niemann-Pick disease type C symptomatology: an expert-based clinical description

(2013) Eugen Mengel et al.   Orphanet Journal of Rare Diseases

Citric acid–γ-cyclodextrin crosslinked oligomers as carriers for doxorubicin delivery

(2013) Resmi Anand et al.   PHOTOCHEMICAL & PHOTOBIOLOGICAL SCIENCES

Iminosugar-based inhibitors of glucosylceramide synthase prolong survival but paradoxically increase brain glucosylceramide levels in Niemann–Pick C mice

(2012) Jennifer B. Nietupski et al.   MOLECULAR GENETICS AND METABOLISM

Environmental applications of water-insoluble β-cyclodextrin–epichlorohydrin polymers

(2012) Nadia Morin-Crini et al.   PROGRESS IN POLYMER SCIENCE

Theranostic Gd(III)-lipid microbubbles for MRI-guided focused ultrasound surgery

(2011) Jameel A. Feshitan et al.   BIOMATERIALS

In vitroandin vivostudies on the complexes of glipizide with water-soluble β-cyclodextrin–epichlorohydrin polymers

(2011) Shufang Nie et al.   DRUG DEVELOPMENT AND INDUSTRIAL PHARMACY

Phase solubility studies and stability of cholesterol/β-cyclodextrin inclusion complexes

(2011) Cristina dos Santos et al.   JOURNAL OF THE SCIENCE OF FOOD AND AGRICULTURE

Niemann-Pick type C disease: molecular mechanisms and potential therapeutic approaches

(2010) Anton I. Rosenbaum et al.   JOURNAL OF NEUROCHEMISTRY

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

Soluble Polymer Carriers for the Treatment of Cancer: The Importance of Molecular Architecture

(2009) Megan E. Fox et al.   ACCOUNTS OF CHEMICAL RESEARCH

Cyclodextrin-Based Polymeric Materials: Synthesis, Properties, and Pharmaceutical/Biomedical Applications

(2009) Frank van de Manakker et al.   BIOMACROMOLECULES

Characteristics of compounds that cross the blood-brain barrier

(2009) William A Banks   BMC Neurology

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