Lysosomal storage diseases and the heat shock response: convergences and therapeutic opportunities

High sphingomyelin levels induce lysosomal damage and autophagy dysfunction in Niemann Pick disease type A

(2014) E Gabandé-Rodríguez et al.   CELL DEATH AND DIFFERENTIATION

Stuck in traffic: an emerging theme in diseases of the nervous system

(2014) Jacques Neefjes et al.   TRENDS IN NEUROSCIENCES

Interplay between autophagy and programmed cell death in mammalian neural stem cells

(2013) Kyung Min Chung et al.   BMB Reports

Co-induction of the heat shock response ameliorates disease progression in a mouse model of human spinal and bulbar muscular atrophy: implications for therapy

(2013) Bilal Malik et al.   BRAIN

Role of endogenous psychosine accumulation in oligodendrocyte differentiation and survival: Implication for Krabbe disease

(2013) Je-Seong Won et al.   BRAIN RESEARCH

Transformation-Associated Changes in Sphingolipid Metabolism Sensitize Cells to Lysosomal Cell Death Induced by Inhibitors of Acid Sphingomyelinase

(2013) Nikolaj H.T. Petersen et al.   CANCER CELL

Regulatory Coordination between Two Major Intracellular Homeostatic Systems

(2013) Karol Dokladny et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Lysosomal cell death at a glance

(2013) S. Aits et al.   JOURNAL OF CELL SCIENCE

Psychosine, the cytotoxic sphingolipid that accumulates in globoid cell leukodystrophy, alters membrane architecture

(2013) Jacqueline A. Hawkins-Salsbury et al.   JOURNAL OF LIPID RESEARCH

Lysosomal Membrane Permeability Stimulates Protein Aggregate Formation in Neurons of a Lysosomal Disease

(2013) M. C. Micsenyi et al.   JOURNAL OF NEUROSCIENCE

The role of autophagy in neurodegenerative disease

(2013) Ralph A Nixon   NATURE MEDICINE

The role of heat shock proteins in Amyotrophic Lateral Sclerosis: The therapeutic potential of Arimoclomol

(2013) Bernadett Kalmar et al.   PHARMACOLOGY & THERAPEUTICS

Alterations in ROS Activity and Lysosomal pH Account for Distinct Patterns of Macroautophagy in LINCL and JNCL Fibroblasts

(2013) José Manuel Vidal-Donet et al.   PLoS One

Histone deacetylase inhibitors increase glucocerebrosidase activity in Gaucher disease by modulation of molecular chaperones

(2013) C. Yang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Celastrol increases glucocerebrosidase activity in Gaucher disease by modulating molecular chaperones

(2013) C. Yang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

TLR4-MD-2 complex is negatively regulated by an endogenous ligand, globotetraosylceramide

(2013) Y. Kondo et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Emerging therapies and therapeutic concepts for lysosomal storage diseases

(2013) Thomas Kirkegaard   Expert Opinion on Orphan Drugs

Lysosomal dysfunction causes neurodegeneration in mucolipidosis II ‘knock-in’ mice

(2012) K. Kollmann et al.   BRAIN

Contribution of brain inflammation to neuronal cell death in neuronopathic forms of Gaucher's disease

(2012) Einat B. Vitner et al.   BRAIN

Pathogenesis and therapy of inclusion body myositis

(2012) Steven A. Greenberg   CURRENT OPINION IN NEUROLOGY

Lysosomal storage disorders: The cellular impact of lysosomal dysfunction

(2012) Frances M. Platt et al.   JOURNAL OF CELL BIOLOGY

Sphingosine mediates TNFα-induced lysosomal membrane permeabilization and ensuing programmed cell death in hepatoma cells

(2012) Chiara Ullio et al.   JOURNAL OF LIPID RESEARCH

Sphingolipids: regulators of crosstalk between apoptosis and autophagy

(2012) Megan M. Young et al.   JOURNAL OF LIPID RESEARCH

The therapeutic potential of pharmacological chaperones and proteosomal inhibitors, Celastrol and MG132 in the treatment of sialidosis

(2012) Erin M. O'Leary et al.   MOLECULAR GENETICS AND METABOLISM

Hsp72 preserves muscle function and slows progression of severe muscular dystrophy

(2012) Stefan M. Gehrig et al.   NATURE

Sensitivity to Lysosome-Dependent Cell Death Is Directly Regulated by Lysosomal Cholesterol Content

(2012) Hanna Appelqvist et al.   PLoS One

Chaperone-mediated autophagy: a unique way to enter the lysosome world

(2012) Susmita Kaushik et al.   TRENDS IN CELL BIOLOGY

Lipid storage disorders block lysosomal trafficking by inhibiting a TRP channel and lysosomal calcium release

(2012) Dongbiao Shen et al.   Nature Communications

Regulation of HSF1 Function in the Heat Stress Response: Implications in Aging and Disease

(2011) Julius Anckar et al.   Annual Review of Biochemistry

Peripheral Neuropathy in the Twitcher Mouse Involves the Activation of Axonal Caspase 3

(2011) Benjamin Smith et al.   ASN Neuro

Myelination in the absence of UDP-galactose:ceramide galactosyl-transferase and fatty acid 2 -hydroxylase

(2011) Marion Meixner et al.   BMC NEUROSCIENCE

TRPML: Transporters of metals in lysosomes essential for cell survival?

(2011) Kirill Kiselyov et al.   CELL CALCIUM

GD1a modulates GM-CSF-induced cell proliferation

(2011) A.X.S. Santos et al.   CYTOKINE

BAG3 mediates chaperone-based aggresome-targeting and selective autophagy of misfolded proteins

(2011) Martin Gamerdinger et al.   EMBO REPORTS

Intracerebroventricular enzyme infusion corrects central nervous system pathology and dysfunction in a mouse model of metachromatic leukodystrophy

(2011) S. Stroobants et al.   HUMAN MOLECULAR GENETICS

Hsp70.1 and related lysosomal factors for necrotic neuronal death

(2011) Tetsumori Yamashima   JOURNAL OF NEUROCHEMISTRY

Unesterified Cholesterol Accumulation in Late Endosomes/Lysosomes Causes Neurodegeneration and Is Prevented by Driving Cholesterol Export from This Compartment

(2011) A. Aqul et al.   JOURNAL OF NEUROSCIENCE

The cellular pathology of lysosomal diseases

(2011) Timothy M Cox et al.   JOURNAL OF PATHOLOGY

Higher apoptotic state in Fabry disease peripheral blood mononuclear cells.

(2011) Pablo N. De Francesco et al.   MOLECULAR GENETICS AND METABOLISM

Molecular chaperones in protein folding and proteostasis

(2011) F. Ulrich Hartl et al.   NATURE

Histone deacetylase inhibitors prevent the degradation and restore the activity of glucocerebrosidase in Gaucher disease

(2011) J. Lu et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

Calpain-mediated Hsp70.1 cleavage in hippocampal CA1 neuronal death

(2010) Shunya Sahara et al.   BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS

HSP70 and lysosomal storage disorders: novel therapeutic opportunities

(2010) Nikolaj H.T. Petersen et al.   BIOCHEMICAL SOCIETY TRANSACTIONS

Plasma globotriaosylsphingosine: Diagnostic value and relation to clinical manifestations of Fabry disease

(2010) S.M. Rombach et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE

GT1b-induced neurotoxicity is mediated by the Akt/GSK-3/tau signaling pathway but not caspase-3 in mesencephalic dopaminergic neurons

(2010) Eun S Chung et al.   BMC NEUROSCIENCE

Interleukin-2 gene transfer potentiates the α-galactosylceramide-stimulated antitumor effect by the induction of TRAIL in NKT and NK cells in mouse models of subcutaneous and metastatic carcinoma

(2010) Yoshiki Nishihori et al.   CANCER BIOLOGY & THERAPY

Connecting Hsp70, sphingolipid metabolism and lysosomal stability

(2010) Nikolaj H.T. Petersen et al.   CELL CYCLE

Chemical and/or biological therapeutic strategies to ameliorate protein misfolding diseases

(2010) Derrick Sek Tong Ong et al.   CURRENT OPINION IN CELL BIOLOGY

Ceramide-rich platforms in transmembrane signaling

(2010) Branka Stancevic et al.   FEBS LETTERS

Common and Uncommon Pathogenic Cascades in Lysosomal Storage Diseases

(2010) Einat B. Vitner et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Evidence of lysosomal membrane permeabilization in mucopolysaccharidosis type I: Rupture of calcium and proton homeostasis

(2010) Vanessa Gonçalves Pereira et al.   JOURNAL OF CELLULAR PHYSIOLOGY

Multi-system disorders of glycosphingolipid and ganglioside metabolism

(2010) You-Hai Xu et al.   JOURNAL OF LIPID RESEARCH

Protein Quality Control in the Cytosol and the Endoplasmic Reticulum: Brothers in Arms

(2010) Alexander Buchberger et al.   MOLECULAR CELL

Hsp70 stabilizes lysosomes and reverts Niemann–Pick disease-associated lysosomal pathology

(2010) Thomas Kirkegaard et al.   NATURE

Lipids on Trial: The Search for the Offending Metabolite in Niemann-Pick type C Disease

(2010) Emyr Lloyd-Evans et al.   TRAFFIC

Improvement in Lipid and Protein Trafficking in Niemann-Pick C1 Cells by Correction of a Secondary Enzyme Defect

(2010) Cecilia Devlin et al.   TRAFFIC

Protein Folding in the Cytoplasm and the Heat Shock Response

(2010) R. M. Vabulas et al.   Cold Spring Harbor Perspectives in Biology

Activation of Mutant Enzyme Function In Vivo by Proteasome Inhibitors and Treatments that Induce Hsp70

(2010) Laishram R. Singh et al.   PLoS Genetics

Increased Activity and Altered Subcellular Distribution of Lysosomal Enzymes Determine Neuronal Vulnerability in Niemann-Pick Type C1-Deficient Mice

(2009) Asha Amritraj et al.   AMERICAN JOURNAL OF PATHOLOGY

Redox Activity Within the Lysosomal Compartment: Implications for Aging and Apoptosis

(2009) Tino Kurz et al.   ANTIOXIDANTS & REDOX SIGNALING

GD3, an Overexpressed Tumor-Derived Ganglioside, Mediates the Apoptosis of Activated but not Resting T Cells

(2009) G. Sa et al.   CANCER RESEARCH

Lysosomal degradation of membrane lipids

(2009) Thomas Kolter et al.   FEBS LETTERS

Brain gangliosides in axon-myelin stability and axon regeneration

(2009) Ronald L. Schnaar   FEBS LETTERS

HSF1-mediated BAG3 Expression Attenuates Apoptosis in 4-Hydroxynonenal-treated Colon Cancer Cells via Stabilization of Anti-apoptotic Bcl-2 Proteins

(2009) Aaron T. Jacobs et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Ceramide and neurodegeneration: Susceptibility of neurons and oligodendrocytes to cell damage and death

(2009) Arundhati Jana et al.   JOURNAL OF THE NEUROLOGICAL SCIENCES

Heat-Shock Factor 1 Controls Genome-wide Acetylation in Heat-shocked Cells

(2009) Sabrina Fritah et al.   MOLECULAR BIOLOGY OF THE CELL

Transcriptional induction of GRP78/BiP by histone deacetylase inhibitors and resistance to histone deacetylase inhibitor-induced apoptosis

(2009) P. Baumeister et al.   MOLECULAR CANCER THERAPEUTICS

GM1-Ganglioside Accumulation at the Mitochondria-Associated ER Membranes Links ER Stress to Ca2+-Dependent Mitochondrial Apoptosis

(2009) Renata Sano et al.   MOLECULAR CELL

NAADP mobilizes calcium from acidic organelles through two-pore channels

(2009) Peter J. Calcraft et al.   NATURE

Lysosome biogenesis and lysosomal membrane proteins: trafficking meets function

(2009) Paul Saftig et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

Converging concepts of protein folding in vitro and in vivo

(2009) F Ulrich Hartl et al.   NATURE STRUCTURAL & MOLECULAR BIOLOGY

GD3 Synthase Overexpression Sensitizes Hepatocarcinoma Cells to Hypoxia and Reduces Tumor Growth by Suppressing the cSrc/NF-κB Survival Pathway

(2009) Josep M. Lluis et al.   PLoS One

Mutations in the Fatty Acid 2-Hydroxylase Gene Are Associated with Leukodystrophy with Spastic Paraparesis and Dystonia

(2008) Simon Edvardson et al.   AMERICAN JOURNAL OF HUMAN GENETICS

Lysosomal involvement in cell death and cancer

(2008) Thomas Kirkegaard et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR CELL RESEARCH

Secondary lipid accumulation in lysosomal disease

(2008) Steven U. Walkley et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR CELL RESEARCH

Lysosomal disorders: From storage to cellular damage

(2008) Andrea Ballabio et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR CELL RESEARCH

Sensitization to the Lysosomal Cell Death Pathway by Oncogene-Induced Down-regulation of Lysosome-Associated Membrane Proteins 1 and 2

(2008) N. Fehrenbacher et al.   CANCER RESEARCH

Chemical and Biological Approaches Synergize to Ameliorate Protein-Folding Diseases

(2008) Ting-Wei Mu et al.   CELL

Hsp70 and cardiac surgery: molecular chaperone and inflammatory regulator with compartmentalized effects

(2008) Petrus R. de Jong et al.   CELL STRESS & CHAPERONES

Suppression of sphingomyelin synthase 1 by small interference RNA is associated with enhanced ceramide production and apoptosis after photodamage

(2008) Duska Separovic et al.   EXPERIMENTAL CELL RESEARCH

The unexpected role of acid sphingomyelinase in cell death and the pathophysiology of common diseases

(2008) Eric L. Smith et al.   FASEB JOURNAL

Proteotoxic stress and inducible chaperone networks in neurodegenerative disease and aging

(2008) R. I. Morimoto   GENES & DEVELOPMENT

Fusogenicity of membranes: The impact of acid sphingomyelinase on innate immune responses

(2008) Olaf Utermöhlen et al.   IMMUNOBIOLOGY

Oxidative stress in NPC1 deficient cells: protective effect of allopregnanolone

(2008) Stefania Zampieri et al.   JOURNAL OF CELLULAR AND MOLECULAR MEDICINE

Globotriaosylceramide induces oxidative stress and up-regulates cell adhesion molecule expression in Fabry disease endothelial cells

(2008) Jin-Song Shen et al.   MOLECULAR GENETICS AND METABOLISM

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

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

Elevated globotriaosylsphingosine is a hallmark of Fabry disease

(2008) J. M. Aerts et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Partial Restoration of Mutant Enzyme Homeostasis in Three Distinct Lysosomal Storage Disease Cell Lines by Altering Calcium Homeostasis

(2008) Ting-Wei Mu et al.   PLOS BIOLOGY