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Adeno-associated viral vector serotype 9–based gene therapy for Niemann-Pick disease type A.
- Published in:
- Science Translational Medicine, 2019, v. 11, n. 506, p. 1, doi. 10.1126/scitranslmed.aat3738
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- Article
New paradigms for the treatment of lysosomal storage diseases: targeting the endocannabinoid system as a therapeutic strategy.
- Published in:
- 2021
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- Publication type:
- journal article
Merits of Combination Cortical, Subcortical, and Cerebellar Injections for the Treatment of Niemann-Pick Disease Type A.
- Published in:
- Molecular Therapy, 2012, v. 20, n. 10, p. 1893, doi. 10.1038/mt.2012.118
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- Publication type:
- Article
Acid Sphingomyelinase Overexpression Enhances the Antineoplastic Effects of Irradiation In Vitro and In Vivo.
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- Molecular Therapy, 2008, v. 16, n. 9, p. 1565, doi. 10.1038/mt.2008.145
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- Publication type:
- Article
Control of Endothelial Targeting and Intracellular Delivery of Therapeutic Enzymes by Modulating the Size and Shape of ICAM-1-targeted Carriers.
- Published in:
- Molecular Therapy, 2008, v. 16, n. 8, p. 1450, doi. 10.1038/mt.2008.127
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- Publication type:
- Article
417. Combination Brain and Systemic Injections of AAV Results in Whole Body Therapy and Extension of Lifespan in the Niemann-Pick Mouse.
- Published in:
- Molecular Therapy, 2006, v. 13, p. S160, doi. 10.1016/j.ymthe.2006.08.481
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- Publication type:
- Article
Identification of Novel Biomarkers for Niemann–Pick Disease Using Gene Expression Analysis of Acid Sphingomyelinase Knockout Mice.
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- Molecular Therapy, 2006, v. 13, n. 3, p. 556, doi. 10.1016/j.ymthe.2005.08.020
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- Publication type:
- Article
Lysosomal Enzyme Delivery by ICAM-1-Targeted Nanocarriers Bypassing Glycosylation- and Clathrin-Dependent Endocytosis.
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- Molecular Therapy, 2006, v. 13, n. 1, p. 135, doi. 10.1016/j.ymthe.2005.07.687
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- Publication type:
- Article
AAV8-Mediated Hepatic Expression of Acid Sphingomyelinase Corrects the Metabolic Defect in the Visceral Organs of a Mouse Model of Niemann–Pick Disease
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- Molecular Therapy, 2005, v. 12, n. 3, p. 431, doi. 10.1016/j.ymthe.2005.03.011
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- Publication type:
- Article
AAV Vector-Mediated Correction of Brain Pathology in a Mouse Model of Niemann–Pick A Disease
- Published in:
- Molecular Therapy, 2005, v. 11, n. 5, p. 754, doi. 10.1016/j.ymthe.2005.01.011
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- Publication type:
- Article
Gene therapy for genetic diseases.
- Published in:
- Pediatrics International, 1998, v. 40, n. 3, p. 191, doi. 10.1111/j.1442-200X.1998.tb01912.x
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- Publication type:
- Article
Identification of a Novel Acid Sphingomyelinase Activity Associated with Recombinant Human Acid Ceramidase.
- Published in:
- Biomolecules (2218-273X), 2023, v. 13, n. 11, p. 1623, doi. 10.3390/biom13111623
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- Publication type:
- Article
N-AS-triggered SPMs are direct regulators of microglia in a model of Alzheimer's disease.
- Published in:
- Nature Communications, 2020, v. 11, n. 1, p. 1, doi. 10.1038/s41467-020-16080-4
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- Publication type:
- Article
Acid sphingomyelinase activity triggers microparticle release from glial cells.
- Published in:
- 2009
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- Publication type:
- Erratum
Acid sphingomyelinase activity triggers microparticle release from glial cells.
- Published in:
- EMBO Journal, 2009, v. 28, n. 8, p. 1043, doi. 10.1038/emboj.2009.45
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- Publication type:
- Article
Loss of acid ceramidase in myeloid cells suppresses intestinal neutrophil recruitment.
- Published in:
- FASEB Journal, 2018, v. 32, n. 5, p. 2339, doi. 10.1096/fj.201700585R
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- Publication type:
- Article
Acid ceramidase improves the quality of oocytes and embryos and the outcome of in vitro fertilization.
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- FASEB Journal, 2010, v. 24, n. 4, p. 1229, doi. 10.1096/fj.09-145508
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- Publication type:
- Article
The unexpected role of acid sphingomyelinase in cell death and the pathophysiology of common diseases.
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- FASEB Journal, 2008, v. 22, n. 10, p. 3419, doi. 10.1096/fj.08-108043
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- Publication type:
- Article
Acid ceramidase is a novel factor required for early embryo survival.
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- FASEB Journal, 2007, v. 21, n. 7, p. 1403, doi. 10.1096/fj.06-7016com
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- Publication type:
- Article
Recombinant Acid Ceramidase Reduces Inflammation and Infection in Cystic Fibrosis.
- Published in:
- 2020
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- Publication type:
- journal article
Preimplantation genetic diagnosis for Niemann-Pick disease type B.
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- Prenatal Diagnosis, 2004, v. 24, n. 12, p. 943, doi. 10.1002/pd.1050
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- Publication type:
- Article
SiO<sub>2</sub> stimulates macrophage stress to induce the transformation of lung fibroblasts into myofibroblasts and its relationship with the sphingomyelin metabolic pathway.
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- Journal of Applied Toxicology, 2021, v. 41, n. 10, p. 1584, doi. 10.1002/jat.4148
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- Publication type:
- Article
The clinical spectrum of SMA‐PME and in vitro normalization of its cellular ceramide profile.
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- Annals of Clinical & Translational Neurology, 2022, v. 9, n. 12, p. 1941, doi. 10.1002/acn3.51687
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- Publication type:
- Article
Sphingomyelin 16:0 is a therapeutic target for neuronal death in acid sphingomyelinase deficiency.
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- Cell Death & Disease, 2023, v. 14, n. 4, p. 1, doi. 10.1038/s41419-023-05784-2
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- Publication type:
- Article
Sphingoid long chain bases prevent lung infection by Pseudomonas aeruginosa.
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- EMBO Molecular Medicine, 2014, v. 6, n. 9, p. 1205, doi. 10.15252/emmm.201404075
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- Publication type:
- Article
Pharmacological reversion of sphingomyelin-induced dendritic spine anomalies in a Niemann Pick disease type A mouse model.
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- EMBO Molecular Medicine, 2014, v. 6, n. 3, p. 398, doi. 10.1002/emmm.201302649
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- Publication type:
- Article
Systemic ceramide accumulation leads to severe and varied pathological consequences.
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- EMBO Molecular Medicine, 2013, v. 5, n. 6, p. 827, doi. 10.1002/emmm.201202301
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- Publication type:
- Article
The sphingosine 1-phosphate receptor 1 causes tissue retention by inhibiting the entry of peripheral tissue T lymphocytes into afferent lymphatics.
- Published in:
- Nature Immunology, 2008, v. 9, n. 1, p. 42, doi. 10.1038/ni1534
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- Publication type:
- Article
Quantitative Systems Pharmacology Modeling of Acid Sphingomyelinase Deficiency and the Enzyme Replacement Therapy Olipudase Alfa Is an Innovative Tool for Linking Pathophysiology and Pharmacology.
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- CPT: Pharmacometrics & Systems Pharmacology, 2018, v. 7, n. 7, p. 442, doi. 10.1002/psp4.12304
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- Publication type:
- Article
Acid sphingomyelinase, cell membranes and human disease: Lessons from Niemann–Pick disease
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- FEBS Letters, 2010, v. 584, n. 9, p. 1895, doi. 10.1016/j.febslet.2009.11.083
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- Publication type:
- Article
Signalling Effects Induced by Acid Ceramidase in Human Epithelial Or Leukemic Cell Lines.
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- Cellular Physiology & Biochemistry (Cell Physiol Biochem Press GmbH & Co. KG), 2019, v. 52, n. 5, p. 1092, doi. 10.33594/000000074
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- Publication type:
- Article
Improvement in Lipid and Protein Trafficking in Niemann-Pick C1 Cells by Correction of a Secondary Enzyme Defect.
- Published in:
- Traffic, 2010, v. 11, n. 5, p. 601, doi. 10.1111/j.1600-0854.2010.01046.x
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- Publication type:
- Article
Neuronal SphK1 acetylates COX2 and contributes to pathogenesis in a model of Alzheimer’s Disease.
- Published in:
- Nature Communications, 2018, v. 9, n. 1, p. 1, doi. 10.1038/s41467-018-03674-2
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- Publication type:
- Article
Pathological roles of the VEGF/SphK pathway in Niemann-Pick type C neurons.
- Published in:
- Nature Communications, 2014, v. 5, n. 11, p. 5514, doi. 10.1038/ncomms6514
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- Publication type:
- Article
Dose Responsive Effects of Subcutaneous Pentosan Polysulfate Injection in Mucopolysaccharidosis Type VI Rats and Comparison to Oral Treatment.
- Published in:
- PLoS ONE, 2014, v. 9, n. 6, p. 1, doi. 10.1371/journal.pone.0100882
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- Publication type:
- Article
Recombinant Human Acid Sphingomyelinase as an Adjuvant to Sorafenib Treatment of Experimental Liver Cancer
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- PLoS ONE, 2013, v. 8, n. 5, p. 1, doi. 10.1371/journal.pone.0065620
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- Publication type:
- Article
Acid Ceramidase Maintains the Chondrogenic Phenotype of Expanded Primary Chondrocytes and Improves the Chondrogenic Differentiation of Bone Marrow-Derived Mesenchymal Stem Cells.
- Published in:
- PLoS ONE, 2013, v. 8, n. 4, p. 1, doi. 10.1371/journal.pone.0062715
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- Publication type:
- Article
Pentosan Polysulfate: A Novel Therapy for the Mucopolysaccharidoses.
- Published in:
- PLoS ONE, 2013, v. 8, n. 1, p. 1, doi. 10.1371/journal.pone.0054459
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- Publication type:
- Article
Anti-TNF-Alpha Therapy Enhances the Effects of Enzyme Replacement Therapy in Rats with Mucopolysaccharidosis Type VI.
- Published in:
- PLoS ONE, 2011, v. 6, n. 8, p. 1, doi. 10.1371/journal.pone.0022447
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- Publication type:
- Article
Niemann-Pick disease type C1 is a sphingosine storage disease that causes deregulation of lysosomal calcium.
- Published in:
- Nature Medicine, 2008, v. 14, n. 11, p. 1247, doi. 10.1038/nm.1876
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- Publication type:
- Article
Oocyte apoptosis is suppressed by disruption of the acid sphingomyelinase gene or by sphingosine -1-phosphate therapy.
- Published in:
- Nature Medicine, 2000, v. 6, n. 10, p. 1109, doi. 10.1038/80442
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- Publication type:
- Article
Ex vivo gene therapy using bone marrow-derived cells: combined effects of intracerebral and intravenous transplantation in a mouse model of niemann–pick disease
- Published in:
- Molecular Therapy, 2003, v. 8, n. 6, p. 876, doi. 10.1016/j.ymthe.2003.07.008
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- Publication type:
- Article
Safety Study of Sodium Pentosan Polysulfate for Adult Patients with Mucopolysaccharidosis Type II.
- Published in:
- Diagnostics (2075-4418), 2019, v. 9, n. 4, p. 226, doi. 10.3390/diagnostics9040226
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- Publication type:
- Article
Identification of a 3′ acceptor splice site mutation (g2610c) in the acid sphingomyelinase gene of patients with Niemann - Pick disease.
- Published in:
- Human Molecular Genetics, 1993, v. 2, n. 2, p. 205
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- Publication type:
- Article
The cholesterol transporter NPC1 is essential for epigenetic regulation and maturation of oligodendrocyte lineage cells.
- Published in:
- Nature Communications, 2023, v. 14, n. 1, p. 1, doi. 10.1038/s41467-023-39733-6
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- Publication type:
- Article
Ceramide and Ischemia/Reperfusion Injury.
- Published in:
- Journal of Lipids, 2018, p. 1, doi. 10.1155/2018/3646725
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- Publication type:
- Article
Patterned cerebellar Purkinje cell death in a transgenic mouse model of Niemann Pick type A/B disease.
- Published in:
- European Journal of Neuroscience, 2001, v. 13, n. 10, p. 1873, doi. 10.1046/j.0953-816X.2001.01564.x
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- Publication type:
- Article
Structural, compositional, and biomechanical alterations of the lumbar spine in rats with mucopolysaccharidosis type VI (Maroteaux-Lamy syndrome).
- Published in:
- Journal of Orthopaedic Research, 2013, v. 31, n. 4, p. 621, doi. 10.1002/jor.22281
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- Publication type:
- Article
The molecular medicine of acid ceramidase.
- Published in:
- Biological Chemistry, 2015, v. 396, n. 6/7, p. 759, doi. 10.1515/hsz-2014-0290
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- Publication type:
- Article
Using whole-exome sequencing to investigate the genetic bases of lysosomal storage diseases of unknown etiology.
- Published in:
- Human Mutation, 2017, v. 38, n. 11, p. 1491, doi. 10.1002/humu.23291
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- Publication type:
- Article