Ernesto R. Bongarzone

Secondary accumulation of lyso-platelet activating factors in lysosomal storage diseases

Deficiency of galactosyl-ceramidase in adult oligodendrocytes worsens disease severity during chronic experimental allergic encephalomyelitis

Hyperglycemia increases SCO-spondin and Wnt5a secretion into the cerebrospinal fluid to regulate ependymal cell beating and glucose sensing

Tumor-targeting cell-penetrating peptide, p28, for glioblastoma imaging and therapy

Preclinical studies in Krabbe disease: A model for the investigation of novel combination therapies for lysosomal storage diseases

The Pathogenic Sphingolipid Psychosine is Secreted in Extracellular Vesicles in the Brain of a Mouse Model of Krabbe Disease

Krabbe disease: New hope for an old disease

Waning efficacy in a long-term AAV-mediated gene therapy study in the murine model of Krabbe disease

Synaptic Function and Dysfunction in Lysosomal Storage Diseases

Astrocyte Support for Oligodendrocyte Differentiation can be Conveyed via Extracellular Vesicles but Diminishes with Age

Krabbe disease successfully treated via monotherapy of intrathecal gene therapy

Macrophages Expressing GALC Improve Peripheral Krabbe Disease by a Mechanism Independent of Cross-Correction

Brainstem development requires galactosylceramidase and is critical for pathogenesis in a model of Krabbe disease

Lead Optimization of Benzoxazolone Carboxamides as Orally Bioavailable and CNS Penetrant Acid Ceramidase Inhibitors

Mass spectrometry imaging and LC/MS reveal decreased cerebellar phosphoinositides in Niemann-Pick type C1-null mice

Deregulation of signalling in genetic conditions affecting the lysosomal metabolism of cholesterol and galactosyl-sphingolipids

Intrathecal administration of AAV/GALC vectors in 10-11-day-old twitcher mice improves survival and is enhanced by bone marrow transplant: Intrathecal AAV Combined With BMT To Treat Krabbe Disease

Hematopoietic Stem cell transplantation and lentiviral vector-based gene therapy for Krabbe's disease: Present convictions and future prospects: BMT-Lentiviral Vectors Therapy for Krabbe's Disease

Extracellular vesicle fibrinogen induces encephalitogenic CD8+ T cells in a mouse model of multiple sclerosis

Inhibition of IGF-1-PI3K-Akt-mTORC2 in lipid rafts increases neuronal vulnerability in a genetic lysosomal glycosphingolipidosis

The Role of Vesicle Trafficking and Release in Oligodendrocyte Biology

Standard-Flow LC and Thermal Focusing ESI Elucidates Altered Liver Proteins in Late Stage Niemann–Pick, Type C1 Disease

AAVrh10 Gene Therapy Ameliorates Central and Peripheral Nervous System Disease in Canine Globoid Cell Leukodystrophy (Krabbe Disease)

Long-Term Improvement of Neurological Signs and Metabolic Dysfunction in a Mouse Model of Krabbe’s Disease after Global Gene Therapy

α-Synuclein interacts directly but reversibly with psychosine: implications for α-synucleinopathies

Analysis of age-related changes in psychosine metabolism in the human brain

Psychosine remodels model lipid membranes at neutral pH

microRNA-219 Reduces Viral Load and Pathologic Changes in Theiler's Virus-Induced Demyelinating Disease

miR-219 Cooperates with miR-338 in Myelination and Promotes Myelin Repair in the CNS

Psychosine enhances the shedding of membrane microvesicles: Implications in demyelination in Krabbe’s disease