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

PEGylation as a strategy for improving nanoparticle-based drug and gene delivery

(2016) Jung Soo Suk et al.   ADVANCED DRUG DELIVERY REVIEWS

LDL–cholesterol transport to the endoplasmic reticulum

(2016) Simon G. Pfisterer et al.   CURRENT OPINION IN LIPIDOLOGY

Materials for non-viral intracellular delivery of messenger RNA therapeutics

(2016) Kevin J. Kauffman et al.   JOURNAL OF CONTROLLED RELEASE

A Comparative Study on the Alterations of Endocytic Pathways in Multiple Lysosomal Storage Disorders

(2016) Jeff Rappaport et al.   MOLECULAR PHARMACEUTICS

Chronic administration of an HDAC inhibitor treats both neurological and systemic Niemann-Pick type C disease in a mouse model

(2016) Md. Suhail Alam et al.   Science 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

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

(2016) Satoe Takahashi et al.   Scientific Reports

DSPE-PEG: A Distinctive Component in Drug Delivery System

(2015) Jing Che et al.   CURRENT PHARMACEUTICAL DESIGN

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

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

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

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

Glycosylation inhibition reduces cholesterol accumulation in NPC1 protein-deficient cells

(2015) Jian Li et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

Niemann-Pick C1 Affects the Gene Delivery Efficacy of Degradable Polymeric Nanoparticles

(2014) Ahmed A. Eltoukhy et al.   ACS Nano

Restarting stalled autophagy a potential therapeutic approach for the lipid storage disorder, Niemann-Pick type C1 disease

(2014) Sovan Sarkar et al.   Autophagy

Collaborative Development of 2-Hydroxypropyl-β-Cyclodextrin for the Treatment of Niemann-Pick Type C1 Disease

(2014) Elizabeth Ottinger et al.   CURRENT TOPICS IN MEDICINAL CHEMISTRY

PEG–lipid micelles as drug carriers: physiochemical attributes, formulation principles and biological implication

(2014) Kanwaldeep K. Gill et al.   JOURNAL OF DRUG TARGETING

Development and validation of sensitive LC-MS/MS assays for quantification of HP-β-CD in human plasma and CSF

(2014) Hui Jiang et al.   JOURNAL OF LIPID RESEARCH

Non-viral vectors for gene-based therapy

(2014) Hao Yin et al.   NATURE REVIEWS GENETICS

Lipopeptide nanoparticles for potent and selective siRNA delivery in rodents and nonhuman primates

(2014) Y. Dong et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Therapeutic bispecific antibodies cross the blood-brain barrier in nonhuman primates

(2014) Y. J. Yu et al.   Science Translational Medicine

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

Factors Influencing the Surface Modification of Mesenchymal Stem Cells with Fluorescein-Pegylated Lipids

(2013) Yoshimasa Takafuji et al.   BIOLOGICAL & PHARMACEUTICAL BULLETIN

Synthesis of 2-Hydroxypropyl-β-cyclodextrin/Pluronic-Based Polyrotaxanes via Heterogeneous Reaction as Potential Niemann-Pick Type C Therapeutics

(2013) Yawo A. Mondjinou et al.   BIOMACROMOLECULES

Efficiency of siRNA delivery by lipid nanoparticles is limited by endocytic recycling

(2013) Gaurav Sahay et al.   NATURE BIOTECHNOLOGY

Treatment of Niemann–Pick Type C Disease by Histone Deacetylase Inhibitors

(2013) Paul Helquist et al.   Neurotherapeutics

Transcytosis and brain uptake of transferrin-containing nanoparticles by tuning avidity to transferrin receptor

(2013) D. T. Wiley et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

(2013) Sovan Sarkar et al.   Cell Reports

Liposomal drug delivery systems: From concept to clinical applications

(2012) Theresa M. Allen et al.   ADVANCED DRUG DELIVERY REVIEWS

Autophagy in lysosomal storage disorders

(2012) Andrew P. Lieberman et al.   Autophagy

Stimulated Release of Cholesterol from Liposomal Membranes by a PEGylated Phospholipid

(2012) Vaclav Janout et al.   BIOCONJUGATE CHEMISTRY

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

PEGylated PRINT Nanoparticles: The Impact of PEG Density on Protein Binding, Macrophage Association, Biodistribution, and Pharmacokinetics

(2012) Jillian L. Perry et al.   NANO LETTERS

A Dense Poly(Ethylene Glycol) Coating Improves Penetration of Large Polymeric Nanoparticles Within Brain Tissue

(2012) E. A. Nance et al.   Science Translational Medicine

Recent Advances in Nonviral Vectors for Gene Delivery

(2011) Xia Guo et al.   ACCOUNTS OF CHEMICAL RESEARCH

Histone deacetylase inhibitor treatment dramatically reduces cholesterol accumulation in Niemann-Pick type C1 mutant human fibroblasts

(2011) N. H. Pipalia et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

Cholesterol Pathways Affected by Small Molecules That Decrease Sterol Levels in Niemann-Pick Type C Mutant Cells

(2010) Madalina Rujoi et al.   PLoS One

Tyrosine polyethylene glycol (PEG)-micelle magnetic resonance contrast agent for the detection of lipid rich areas in atherosclerotic plaque

(2009) Anne Beilvert et al.   MAGNETIC RESONANCE IN MEDICINE

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

Pluronic block copolymers: Evolution of drug delivery concept from inert nanocarriers to biological response modifiers

(2008) Elena V. Batrakova et al.   JOURNAL OF CONTROLLED RELEASE

Cellular cholesterol trafficking and compartmentalization

(2008) Elina Ikonen   NATURE REVIEWS MOLECULAR CELL BIOLOGY