Synthetic high-density lipoprotein nanoparticles for the treatment of Niemann–Pick diseases

Endocytosis of lipoproteins

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Protein kinase C activity is a protective modifier of Purkinje neuron degeneration in cerebellar ataxia

(2018) Ravi Chopra et al.   HUMAN MOLECULAR GENETICS

Modulating membrane fluidity corrects Batten disease phenotypes in vitro and in vivo

(2018) Mark L. Schultz et al.   NEUROBIOLOGY OF DISEASE

Coordinate regulation of mutant NPC1 degradation by selective ER autophagy and MARCH6-dependent ERAD

(2018) Mark L. Schultz et al.   Nature Communications

Intravenous toxicity and toxicokinetics of an HDL mimetic, Fx-5A peptide complex, in cynomolgus monkeys

(2018) Mohammed Bourdi et al.   REGULATORY TOXICOLOGY AND PHARMACOLOGY

Types A and B Niemann-Pick disease

(2017) Edward H. Schuchman et al.   MOLECULAR GENETICS AND METABOLISM

Evaluation of Antisense Oligonucleotides Targeting ATXN3 in SCA3 Mouse Models

(2017) Lauren R. Moore et al.   Molecular Therapy-Nucleic Acids

Rapid intraoperative histology of unprocessed surgical specimens via fibre-laser-based stimulated Raman scattering microscopy

(2017) Daniel A. Orringer et al.   Nature Biomedical Engineering

High-Density Lipoproteins: Nature’s Multifunctional Nanoparticles

(2016) Rui Kuai et al.   ACS Nano

Influence of route of administration and lipidation of apolipoprotein A-I peptide on pharmacokinetics and cholesterol mobilization

(2016) Jie Tang et al.   JOURNAL OF LIPID RESEARCH

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

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ApoA-IMilano phospholipid complex (ETC-216) infusion in human volunteers. Insights into the phenotypic characteristics of ApoA-IMilano carriers

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The effect of phospholipid composition of reconstituted HDL on its cholesterol efflux and anti-inflammatory properties

(2015) Anna Schwendeman et al.   JOURNAL OF LIPID 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

Infusion of Reconstituted High‐Density Lipoprotein, CSL112, in Patients With Atherosclerosis: Safety and Pharmacokinetic Results From a Phase 2a Randomized Clinical Trial

(2015) Pierluigi Tricoci et al.   Journal of the American Heart Association

Effects of the high-density lipoprotein mimetic agent CER-001 on coronary atherosclerosis in patients with acute coronary syndromes: a randomized trial

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

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HDL-targeted therapies: progress, failures and future

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Reconstituted HDL for the acute treatment of acute coronary syndrome

(2013) Brian R. Krause et al.   CURRENT OPINION IN LIPIDOLOGY

Reverse cholesterol transport fluxes

(2013) Marc Hellerstein et al.   CURRENT OPINION IN LIPIDOLOGY

CLN3 Loss Disturbs Membrane Microdomain Properties and Protein Transport in Brain Endothelial Cells

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High-Density Lipoprotein Function, Dysfunction, and Reverse Cholesterol Transport

(2012) Edward A. Fisher et al.   ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY

Ryanodine receptor antagonists adapt NPC1 proteostasis to ameliorate lipid storage in Niemann–Pick type C disease fibroblasts

(2012) Ting Yu 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

The apolipoprotein A-I mimetic peptide ETC-642 exhibits anti-inflammatory properties that are comparable to high density lipoproteins

(2011) Belinda A. Di Bartolo et al.   ATHEROSCLEROSIS

ABCA1-dependent mobilization of lysosomal cholesterol requires functional Niemann–Pick C2 but not Niemann–Pick C1 protein

(2011) Emmanuel Boadu et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR AND CELL BIOLOGY OF LIPIDS

Structure/Function Relationships of Apolipoprotein A-I Mimetic Peptides

(2010) Wilissa D'Souza et al.   CIRCULATION RESEARCH

Amiloride inhibits macropinocytosis by lowering submembranous pH and preventing Rac1 and Cdc42 signaling

(2010) Mirkka Koivusalo et al.   JOURNAL OF CELL BIOLOGY

5A Apolipoprotein Mimetic Peptide Promotes Cholesterol Efflux and Reduces Atherosclerosis in Mice

(2010) M. J. A. Amar et al.   JOURNAL OF PHARMACOLOGY AND EXPERIMENTAL THERAPEUTICS

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

Transport of lipids by ABC proteins: Interactions and implications for cellular toxicity, viability and function

(2009) Irving L.M.H. Aye et al.   CHEMICO-BIOLOGICAL INTERACTIONS

Retroendocytosis pathway of ABCA1/apoA-I contributes to HDL formation

(2009) Yuya Azuma et al.   GENES TO CELLS

Conditional Niemann-Pick C mice demonstrate cell autonomous Purkinje cell neurodegeneration

(2009) Matthew J. Elrick et al.   HUMAN MOLECULAR GENETICS

The National Niemann-Pick Type C1 Disease Database: correlation of lipid profiles, mutations, and biochemical phenotypes

(2009) William S. Garver et al.   JOURNAL OF LIPID RESEARCH

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

Niemann-Pick Type C1 I1061T Mutant Encodes a Functional Protein That Is Selected for Endoplasmic Reticulum-associated Degradation Due to Protein Misfolding

(2008) Mark E. Gelsthorpe et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

NPC2 facilitates bidirectional transfer of cholesterol between NPC1 and lipid bilayers, a step in cholesterol egress from lysosomes

(2008) R. E. Infante et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Label-Free Biomedical Imaging with High Sensitivity by Stimulated Raman Scattering Microscopy

(2008) Christian W. Freudiger et al.   SCIENCE