Image-based screen capturing misfolding status of Niemann-Pick type C1 identifies potential candidates for chaperone drugs

Structural basis for itraconazole-mediated NPC1 inhibition

(2020) Tao Long et al.   Nature Communications

Inter-domain dynamics drive cholesterol transport by NPC1 and NPC1L1 proteins

(2020) Piyali Saha et al.   eLife

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

(2019) Suzanne R. Pfeffer   JOURNAL OF BIOLOGICAL CHEMISTRY

The morphogen Sonic hedgehog inhibits its receptor Patched by a pincer grasp mechanism

(2019) Amalie F. Rudolf et al.   Nature Chemical Biology

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

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

An efficient screening method for purifying and crystallizing membrane proteins using modified clear-native PAGE

(2018) Nanao Suzuki et al.   ANALYTICAL BIOCHEMISTRY

High-content screen for modifiers of Niemann-Pick type C disease in patient cells

(2018) Emily K Pugach et al.   HUMAN MOLECULAR GENETICS

A VCP inhibitor substrate trapping approach (VISTA) enables proteomic profiling of endogenous ERAD substrates

(2018) Edmond Y. Huang et al.   MOLECULAR BIOLOGY OF THE CELL

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

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

Structural Basis for Cholesterol Transport-like Activity of the Hedgehog Receptor Patched

(2018) Yunxiao Zhang et al.   CELL

Simultaneous Targeting of NPC1 and VDAC1 by Itraconazole Leads to Synergistic Inhibition of mTOR Signaling and Angiogenesis

(2016) Sarah A. Head et al.   ACS Chemical Biology

Lysosome and endoplasmic reticulum quality control pathways in Niemann-Pick type C disease

(2016) Mark L. Schultz et al.   BRAIN RESEARCH

Structural Insights into the Niemann-Pick C1 (NPC1)-Mediated Cholesterol Transfer and Ebola Infection

(2016) Xin Gong et al.   CELL

2-Aryl-5-carboxytetrazole as a New Photoaffinity Label for Drug Target Identification

(2016) András Herner et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Structure of human Niemann–Pick C1 protein

(2016) Xiaochun Li et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Triazoles inhibit cholesterol export from lysosomes by binding to NPC1

(2016) Michael N. Trinh et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

The FTMap family of web servers for determining and characterizing ligand-binding hot spots of proteins

(2015) Dima Kozakov et al.   Nature Protocols

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

NPC1L1 is a key regulator of intestinal vitamin K absorption and a modulator of warfarin therapy

(2015) Tappei Takada et al.   Science Translational Medicine

Fomiroid A, a Novel Compound from the Mushroom Fomitopsis nigra, Inhibits NPC1L1-Mediated Cholesterol Uptake via a Mode of Action Distinct from That of Ezetimibe

(2015) Tomohiro Chiba et al.   PLoS One

Structure–activity relationship study of non-steroidal NPC1L1 ligands identified through cell-based assay using pharmacological chaperone effect as a readout

(2014) Fumika Karaki et al.   BIOORGANIC & MEDICINAL CHEMISTRY

Structure–activity relationships of oxysterol-derived pharmacological chaperones for Niemann–Pick type C1 protein

(2014) Kenji Ohgane et al.   BIOORGANIC & MEDICINAL CHEMISTRY LETTERS

Endoplasmic Reticulum-associated Degradation of Niemann-Pick C1

(2014) Naoe Nakasone et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Overcoming Drug Development Bottlenecks With Repurposing: Old drugs learn new tricks

(2014) Stephen M Strittmatter   NATURE MEDICINE

Structure–activity relationship studies of Niemann-Pick type C1-like 1 (NPC1L1) ligands identified by screening assay monitoring pharmacological chaperone effect

(2013) Fumika Karaki et al.   BIOORGANIC & MEDICINAL CHEMISTRY

Discovery of Oxysterol-Derived Pharmacological Chaperones for NPC1: Implication for the Existence of Second Sterol-Binding Site

(2013) Kenji Ohgane et al.   CHEMISTRY & BIOLOGY

Analysis of the phospholipase C-δ1 pleckstrin homology domain using native polyacrylamide gel electrophoresis

(2012) Michikazu Tanio et al.   ANALYTICAL BIOCHEMISTRY

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

Oxysterols are allosteric activators of the oncoprotein Smoothened

(2012) Sigrid Nachtergaele et al.   Nature Chemical Biology

A Fluorescence-Detection Size-Exclusion Chromatography-Based Thermostability Assay for Membrane Protein Precrystallization Screening

(2012) Motoyuki Hattori et al.   STRUCTURE

Ouabain Mimics Low Temperature Rescue of F508del-CFTR in Cystic Fibrosis Epithelial Cells

(2012) Donglei Zhang et al.   Frontiers in Pharmacology

High-resolution Native-PAGE for membrane proteins capable of fluorescence detection and hydrodynamic state evaluation

(2011) Makoto Ihara et al.   ANALYTICAL BIOCHEMISTRY

An “Exacerbate-reverse” Strategy in Yeast Identifies Histone Deacetylase Inhibition as a Correction for Cholesterol and Sphingolipid Transport Defects in Human Niemann-Pick Type C Disease

(2011) Andrew B. Munkacsi et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

The Antifungal Drug Itraconazole Inhibits Vascular Endothelial Growth Factor Receptor 2 (VEGFR2) Glycosylation, Trafficking, and Signaling in Endothelial Cells

(2011) Benjamin A. Nacev et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

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

Amino acid substitution in NPC1 that abolishes cholesterol binding reproduces phenotype of complete NPC1 deficiency in mice

(2011) X. Xie et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809

(2011) F. Van Goor et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

EBImage--an R package for image processing with applications to cellular phenotypes

(2010) G. Pau et al.   BIOINFORMATICS

Identification of Surface Residues on Niemann-Pick C2 Essential for Hydrophobic Handoff of Cholesterol to NPC1 in Lysosomes

(2010) Michael L. Wang et al.   Cell Metabolism

Analysis and Optimization of Copper-Catalyzed Azide–Alkyne Cycloaddition for Bioconjugation

(2009) Vu Hong et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Niemann–Pick C2 (NPC2) and intracellular cholesterol trafficking

(2009) Judith Storch et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR AND CELL BIOLOGY OF LIPIDS

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

(2009) Hyock Joo Kwon et al.   CELL

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