Necroptosis in Niemann–Pick disease, type C1: a potential therapeutic target

High incidence of unrecognized visceral/neurological late-onset Niemann-Pick disease, type C1, predicted by analysis of massively parallel sequencing data sets

(2015) Christopher A. Wassif et al.   GENETICS IN MEDICINE

Adaptations of Energy Metabolism Associated with Increased Levels of Mitochondrial Cholesterol in Niemann-Pick Type C1-deficient Cells

(2014) Barry E. Kennedy et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

RIPK3 as a potential therapeutic target for Gaucher's disease

(2014) Einat B Vitner et al.   NATURE MEDICINE

Improved neuroprotection using miglustat, curcumin and ibuprofen as a triple combination therapy in Niemann–Pick disease type C1 mice

(2014) Ian M. Williams et al.   NEUROBIOLOGY OF DISEASE

Necroptosis Drives Motor Neuron Death in Models of Both Sporadic and Familial ALS

(2014) Diane B. Re et al.   NEURON

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

A novel necroptosis inhibitor—necrostatin-21 and its SAR study

(2013) Zhijie Wu et al.   BIOORGANIC & MEDICINAL CHEMISTRY LETTERS

Efficacy of N-acetylcysteine in phenotypic suppression of mouse models of Niemann–Pick disease, type C1

(2013) Rao Fu et al.   HUMAN MOLECULAR GENETICS

Human and mouse neuroinflammation markers in Niemann-Pick disease, type C1

(2013) Stephanie M. Cologna et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Altered transition metal homeostasis in Niemann–Pick disease, type C1

(2013) Ya Hui Hung et al.   Metallomics

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

(2013) Sovan Sarkar et al.   Cell Reports

Activity and specificity of necrostatin-1, small-molecule inhibitor of RIP1 kinase

(2012) A Degterev et al.   CELL DEATH AND DIFFERENTIATION

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

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

Impaired proteolysis underlies autophagic dysfunction in Niemann–Pick type C disease

(2012) Matthew J. Elrick et al.   HUMAN MOLECULAR GENETICS

Amino acid catabolism: a pivotal regulator of innate and adaptive immunity

(2012) Tracy L. McGaha et al.   IMMUNOLOGICAL REVIEWS

Quantitative Comparison of the Efficacy of Various Compounds in Lowering Intracellular Cholesterol Levels in Niemann-Pick Type C Fibroblasts

(2012) Zachary T. Wehrmann et al.   PLoS One

Necrostatin-1 analogues: critical issues on the specificity, activity and in vivo use in experimental disease models

(2012) N Takahashi et al.   Cell Death & Disease

Molecular definitions of cell death subroutines: recommendations of the Nomenclature Committee on Cell Death 2012

(2011) L Galluzzi et al.   CELL DEATH AND DIFFERENTIATION

Necrostatin-1 ameliorates symptoms in R6/2 transgenic mouse model of Huntington's disease

(2011) S Zhu et al.   Cell Death & Disease

Synergistic protective effects of humanin and necrostatin-1 on hypoxia and ischemia/reperfusion injury

(2010) Xingshun Xu et al.   BRAIN RESEARCH

Necroptosis as an alternative form of programmed cell death

(2010) Dana E Christofferson et al.   CURRENT OPINION IN CELL BIOLOGY

Niemann-Pick disease type C

(2010) Marie T Vanier   Orphanet Journal of Rare Diseases

Miglustat in adult and juvenile patients with Niemann–Pick disease type C: Long-term data from a clinical trial

(2009) James E. Wraith et al.   MOLECULAR GENETICS AND METABOLISM

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

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

Structure–activity relationship study of a novel necroptosis inhibitor, necrostatin-7

(2008) Weihong Zheng et al.   BIOORGANIC & MEDICINAL CHEMISTRY LETTERS

Necrostatin-1 Reduces Histopathology and Improves Functional Outcome after Controlled Cortical Impact in Mice

(2008) Zerong You et al.   JOURNAL OF CEREBRAL BLOOD FLOW AND METABOLISM

Madin-Darby Canine Kidney II Cells: A Pharmacologically Validated System for NPC1L1-Mediated Cholesterol Uptake

(2008) A. B. Weinglass et al.   MOLECULAR PHARMACOLOGY

Identification of RIP1 kinase as a specific cellular target of necrostatins

(2008) Alexei Degterev et al.   Nature Chemical Biology

Niemann-Pick disease type C1 is a sphingosine storage disease that causes deregulation of lysosomal calcium

(2008) Emyr Lloyd-Evans et al.   NATURE MEDICINE