Lysosomal Re-acidification Prevents Lysosphingolipid-Induced Lysosomal Impairment and Cellular Toxicity

Lysosomal Storage Diseases: From Pathophysiology to Therapy

(2015) Giancarlo Parenti et al.   Annual Review of Medicine

Krabbe disease: involvement of connexin43 in the apoptotic effects of sphingolipid psychosine on mouse oligodendrocyte precursors

(2015) A. C. E. Graziano et al.   APOPTOSIS

Neuronopathic lysosomal storage disorders: Approaches to treat the central nervous system

(2015) Maurizio Scarpa et al.   BEST PRACTICE & RESEARCH CLINICAL ENDOCRINOLOGY & METABOLISM

TMEM175 Is an Organelle K + Channel Regulating Lysosomal Function

(2015) Chunlei Cang et al.   CELL

Enzyme replacement therapy of a novel humanized mouse model of globoid cell leukodystrophy

(2015) Frank Matthes et al.   EXPERIMENTAL NEUROLOGY

TNF compromises lysosome acidification and reduces α-synuclein degradation via autophagy in dopaminergic cells

(2015) Mei-Xia Wang et al.   EXPERIMENTAL NEUROLOGY

Galactosylsphingosine (psychosine)-induced demyelination is attenuated by sphingosine 1-phosphate signalling

(2015) C. O'Sullivan et al.   JOURNAL OF CELL SCIENCE

Mechanism of Neuromuscular Dysfunction in Krabbe Disease

(2015) L. Cantuti-Castelvetri et al.   JOURNAL OF NEUROSCIENCE

Mechanism-Based Combination Treatment Dramatically Increases Therapeutic Efficacy in Murine Globoid Cell Leukodystrophy

(2015) J. A. Hawkins-Salsbury et al.   JOURNAL OF NEUROSCIENCE

Psychosine inhibits osteoclastogenesis and bone resorption via G protein-coupled receptor 65

(2015) S. H. Ahn et al.   JOURNAL OF ENDOCRINOLOGICAL INVESTIGATION

Lysosomotropic agents: impact on lysosomal membrane permeabilization and cell death

(2014) Ana M. Villamil Giraldo et al.   BIOCHEMICAL SOCIETY TRANSACTIONS

Correction of lysosomal dysfunction as a therapeutic strategy for neurodegenerative diseases

(2014) Robert E. Boyd et al.   BIOORGANIC & MEDICINAL CHEMISTRY LETTERS

Glucopsychosine increases cytosolic calcium to induce calpain-mediated apoptosis of acute myeloid leukemia cells

(2014) Leonard Angka et al.   CANCER LETTERS

Approaches for detecting lysosomal alkalinization and impaired degradation in fresh and cultured RPE cells: Evidence for a role in retinal degenerations

(2014) Sonia Guha et al.   EXPERIMENTAL EYE RESEARCH

Therapeutic benefit of lentiviral-mediated neonatal intracerebral gene therapy in a mouse model of globoid cell leukodystrophy

(2014) A. Lattanzi et al.   HUMAN MOLECULAR GENETICS

Impairment of homeostasis in lysosomal storage disorders

(2014) Laura Segatori   IUBMB LIFE

Treatment of lysosomal storage disorders: successes and challenges

(2014) Carla E. M. Hollak et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Neuronal inclusions of α-synuclein contribute to the pathogenesis of Krabbe disease

(2014) Benjamin R Smith et al.   JOURNAL OF PATHOLOGY

Sphingolipid lysosomal storage disorders

(2014) Frances M. Platt   NATURE

The voltage-gated sodium channel TPC1 confers endolysosomal excitability

(2014) Chunlei Cang et al.   Nature Chemical Biology

Early axonal loss accompanied by impaired endocytosis, abnormal axonal transport, and decreased microtubule stability occur in the model of Krabbe's disease

(2014) Carla Andreia Teixeira et al.   NEUROBIOLOGY OF DISEASE

Lysosomal alkalization and dysfunction in human fibroblasts with the Alzheimer’s disease-linked presenilin 1 A246E mutation can be reversed with cAMP

(2014) E.E. Coffey et al.   NEUROSCIENCE

Experimental Therapies in the Murine Model of Globoid Cell Leukodystrophy

(2014) Yedda Li et al.   PEDIATRIC NEUROLOGY

The Leukodystrophies

(2014) Hannah Gordon et al.   SEMINARS IN NEUROLOGY

Role of extracellular calcium and mitochondrial oxygen species in psychosine-induced oligodendrocyte cell death

(2014) V Voccoli et al.   Cell Death & Disease

Inhibition of angiogenesis by β-galactosylceramidase deficiency in globoid cell leukodystrophy

(2013) Mirella Belleri 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

Primary Bone Marrow Mesenchymal Stromal Cells Rescue the Axonal Phenotype of Twitcher Mice

(2013) Catarina Oliveira Miranda et al.   CELL TRANSPLANTATION

Beneficial Effects of Lysosome-Modulating and Other Pharmacological and Nanocarrier Agents on Amyloid-beta-treated Cells

(2013) Marie V. Kanazirska et al.   CURRENT PHARMACEUTICAL BIOTECHNOLOGY

Neuronopathic lysosomal storage diseases: Clinical and pathologic findings

(2013) Carlos E. Prada et al.   Developmental Disabilities Research Reviews

MMP-3 mediates psychosine-induced globoid cell formation: Implications for leukodystrophy pathology

(2013) Kumiko Ijichi et al.   GLIA

Neuronal accumulation of glucosylceramide in a mouse model of neuronopathic Gaucher disease leads to neurodegeneration

(2013) T. Farfel-Becker et al.   HUMAN MOLECULAR GENETICS

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

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

The Sphingolipid Psychosine Inhibits Fast Axonal Transport in Krabbe Disease by Activation of GSK3  and Deregulation of Molecular Motors

(2013) L. Cantuti Castelvetri et al.   JOURNAL OF NEUROSCIENCE

Glucosylceramide modulates endolysosomal pH in Gaucher disease

(2013) Dan J. Sillence   MOLECULAR GENETICS AND METABOLISM

The role of autophagy in neurodegenerative disease

(2013) Ralph A Nixon   NATURE MEDICINE

Estrogen receptor β ligand therapy activates PI3K/Akt/mTOR signaling in oligodendrocytes and promotes remyelination in a mouse model of multiple sclerosis

(2013) Shalini Kumar et al.   NEUROBIOLOGY OF DISEASE

CD133/CD140a-Based Isolation of Distinct Human Multipotent Neural Progenitor Cells and Oligodendrocyte Progenitor Cells

(2013) Jing Wang et al.   STEM CELLS AND DEVELOPMENT

Intracellular accumulation of aggregated pyroglutamate amyloid beta: convergence of aging and Aβ pathology at the lysosome

(2012) Line De Kimpe et al.   AGE

Cystic fibrosis transmembrane conductance regulator contributes to reacidification of alkalinized lysosomes in RPE cells

(2012) Ji Liu et al.   AMERICAN JOURNAL OF PHYSIOLOGY-CELL PHYSIOLOGY

Glucocerebrosidase deficiency in substantia nigra of parkinson disease brains

(2012) Matthew E. Gegg et al.   ANNALS OF NEUROLOGY

Enzyme Replacement Therapy for Lysosomal Diseases: Lessons from 20 Years of Experience and Remaining Challenges

(2012) R.J. Desnick et al.   Annual Review of Genomics and Human Genetics

Drosophila TRPML Is Required for TORC1 Activation

(2012) Ching-On Wong et al.   CURRENT BIOLOGY

Oxidative stress as a therapeutic target in globoid cell leukodystrophy

(2012) Jacqueline A. Hawkins-Salsbury et al.   EXPERIMENTAL NEUROLOGY

Treatment options for lysosomal storage disorders: developing insights

(2012) Carin M van Gelder et al.   EXPERT OPINION ON PHARMACOTHERAPY

The galactocerebrosidase enzyme contributes to maintain a functional neurogenic niche during early post-natal CNS development

(2012) Sara Santambrogio et al.   HUMAN MOLECULAR GENETICS

Inhibition of Glycogen Synthase Kinase-3 Ameliorates β-Amyloid Pathology and Restores Lysosomal Acidification and Mammalian Target of Rapamycin Activity in the Alzheimer Disease Mouse Model

(2012) Limor Avrahami et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Loss of Lysosomal Ion Channel Transient Receptor Potential Channel Mucolipin-1 (TRPML1) Leads to Cathepsin B-dependent Apoptosis

(2012) Grace A. Colletti et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Lysosomal storage disorders: The cellular impact of lysosomal dysfunction

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

Stimulation of the D5 dopamine receptor acidifies the lysosomal pH of retinal pigmented epithelial cells and decreases accumulation of autofluorescent photoreceptor debris

(2012) Sonia Guha et al.   JOURNAL OF NEUROCHEMISTRY

Endolysosome involvement in LDL cholesterol-induced Alzheimer's disease-like pathology in primary cultured neurons

(2012) Liang Hui et al.   LIFE SCIENCES

Bone marrow transplantation increases efficacy of central nervous system-directed enzyme replacement therapy in the murine model of globoid cell leukodystrophy

(2012) Elizabeth Y. Qin et al.   MOLECULAR GENETICS AND METABOLISM

Lysosome-dependent pathways as a unifying theme in Parkinson's disease

(2012) George K. Tofaris   MOVEMENT DISORDERS

Lysosome-targeted octadecyl-rhodamine B-liposomes enhance lysosomal accumulation of glucocerebrosidase in Gaucher’s cellsin vitro

(2012) Ritesh Thekkedath et al.   Nanomedicine

Psychosine induces the dephosphorylation of neurofilaments by deregulation of PP1 and PP2A phosphatases

(2012) Ludovico Cantuti-Castelvetri et al.   NEUROBIOLOGY OF DISEASE

Positive Lysosomal Modulation As a Unique Strategy to Treat Age-Related Protein Accumulation Diseases

(2012) Ben A. Bahr et al.   REJUVENATION RESEARCH

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

(2012) Dongbiao Shen et al.   Nature Communications

Axonopathy is a compounding factor in the pathogenesis of Krabbe disease

(2011) Ludovico Cantuti Castelvetri et al.   ACTA NEUROPATHOLOGICA

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

(2011) Benjamin Smith et al.   ASN Neuro

Therapeutic effects of remediating autophagy failure in a mouse model of Alzheimer disease by enhancing lysosomal proteolysis

(2011) Dun-Sheng Yang et al.   Autophagy

Effect of intrastriatal mesenchymal stromal cell injection on progression of a murine model of Krabbe disease

(2011) Shawna E. Wicks et al.   BEHAVIOURAL BRAIN RESEARCH

Proteomic identification of novel targets regulated by the mammalian target of rapamycin pathway during oligodendrocyte differentiation

(2011) William A. Tyler et al.   GLIA

Lysosomal pH and analysis of the counter ion pathways that support acidification

(2011) Jessica E. DiCiccio et al.   JOURNAL OF GENERAL PHYSIOLOGY

Bone Marrow Transplantation Augments the Effect of Brain- and Spinal Cord-Directed Adeno-Associated Virus 2/5 Gene Therapy by Altering Inflammation in the Murine Model of Globoid-Cell Leukodystrophy

(2011) A. S. Reddy et al.   JOURNAL OF NEUROSCIENCE

The cellular pathology of lysosomal diseases

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

CNS-targeted AAV5 gene transfer results in global dispersal of vector and prevention of morphological and function deterioration in CNS of globoid cell leukodystrophy mouse model

(2011) Dar-Shong Lin et al.   MOLECULAR GENETICS AND METABOLISM

Protective Effects of Positive Lysosomal Modulation in Alzheimer's Disease Transgenic Mouse Models

(2011) David Butler et al.   PLoS One

Systemic Delivery of Bone Marrow-Derived Mesenchymal Stromal Cells Diminishes Neuropathology in a Mouse Model of Krabbe's Disease

(2011) Catarina Oliveira Miranda et al.   STEM CELLS

Glycosphingolipid studies of visceral tissues and brain from type 1 Gaucher disease variants

(2010) Olle Nilsson et al.   CLINICAL GENETICS

Altered expression and distribution of cathepsins in neuronopathic forms of Gaucher disease and in other sphingolipidoses

(2010) E. B. Vitner et al.   HUMAN MOLECULAR GENETICS

Widespread enzymatic correction of CNS tissues by a single intracerebral injection of therapeutic lentiviral vector in leukodystrophy mouse models

(2010) Annalisa Lattanzi et al.   HUMAN MOLECULAR GENETICS

Endocannabinoids Prevent β-Amyloid-mediated Lysosomal Destabilization in Cultured Neurons

(2010) Janis Noonan et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

A cation counterflux supports lysosomal acidification

(2010) Benjamin E. Steinberg et al.   JOURNAL OF CELL BIOLOGY

Persistence of psychosine in brain lipid rafts is a limiting factor in the therapeutic recovery of a mouse model for Krabbe disease

(2010) A.B. White et al.   JOURNAL OF NEUROSCIENCE RESEARCH

Proteases and Proteolysis in Alzheimer Disease: A Multifactorial View on the Disease Process

(2010) Bart De Strooper   PHYSIOLOGICAL REVIEWS

Lysosomal Pathology and Osteopetrosis upon Loss of H+-Driven Lysosomal Cl- Accumulation

(2010) S. Weinert et al.   SCIENCE

Identification of Hematopoietic Stem Cell-Specific miRNAs Enables Gene Therapy of Globoid Cell Leukodystrophy

(2010) B. Gentner et al.   Science Translational Medicine

Mesenchymal Lineage Stem Cells Have Pronounced Anti-Inflammatory Effects in the Twitcher Mouse Model of Krabbe's Disease

(2010) Cynthia B. Ripoll et al.   STEM CELLS

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

Unimpaired Lysosomal Acidification in Respiratory Epithelial Cells in Cystic Fibrosis

(2009) Peter M. Haggie et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Disease-causing Mutations in the Cystic Fibrosis Transmembrane Conductance Regulator Determine the Functional Responses of Alveolar Macrophages

(2009) Ludmila V. Deriy et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

IGF-1-stimulated protein synthesis in oligodendrocyte progenitors requires PI3K/mTOR/Akt and MEK/ERK pathways

(2009) Olivia Bibollet-Bahena et al.   JOURNAL OF NEUROCHEMISTRY

Akt Signals through the Mammalian Target of Rapamycin Pathway to Regulate CNS Myelination

(2009) S. P. Narayanan et al.   JOURNAL OF NEUROSCIENCE

Psychosine Accumulates in Membrane Microdomains in the Brain of Krabbe Patients, Disrupting the Raft Architecture

(2009) A. B. White et al.   JOURNAL OF NEUROSCIENCE

Revisiting the Role of Cystic Fibrosis Transmembrane Conductance Regulator and Counterion Permeability in the pH Regulation of Endocytic Organelles

(2009) Herve Barriere et al.   MOLECULAR BIOLOGY OF THE CELL

Significant correction of pathology in brains of twitcher mice following injection of genetically modified mouse neural progenitor cells

(2009) Michelina Strazza et al.   MOLECULAR GENETICS AND METABOLISM

Sensors and regulators of intracellular pH

(2009) Joseph R. Casey et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

Psychosine-induced alterations in peroxisomes of twitcher mouse liver

(2008) Miguel Agustin Contreras et al.   ARCHIVES OF BIOCHEMISTRY AND BIOPHYSICS

The role of AMPK in psychosine mediated effects on oligodendrocytes and astrocytes: implication for Krabbe Disease

(2008) Shailendra Giri et al.   JOURNAL OF NEUROCHEMISTRY

The Cl-/H+ antiporter ClC-7 is the primary chloride permeation pathway in lysosomes

(2008) Austin R. Graves et al.   NATURE

Lysosulfatide Regulates the Motility of a Neural Precursor Cell Line Via Calcium-mediated Process Collapse

(2008) M. Hans et al.   NEUROCHEMICAL RESEARCH

Stimulation of Adenylyl Cyclase and Induction of Brain-Derived Neurotrophic Factor and TrkB mRNA by NKH477, a Novel and Potent Forskolin Derivative

(2003) Shigeru Morinobu et al.   JOURNAL OF NEUROCHEMISTRY