Clinical phenotype of Gaucher disease in relation to properties of mutant glucocerebrosidase in cultured fibroblasts (original) (raw)

Ultrastructural localization of glucocerebrosidase in cultured Gaucher's disease fibroblasts by immunocytochemistry

Arnold Reuser

Journal of Neurology, 1987

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Activity of glucocerebrosidase in extracts of different cell types from type 1 Gaucher disease patients

Sonja Van Weely

Clinical …, 1990

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Role of pH in determining the cell-type-specific residual activity of glucocerebrosidase in type 1 Gaucher disease

Sonja Van Weely

Journal of Clinical Investigation, 1993

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Biosynthesis and maturation of glucocerebrosidase in Gaucher fibroblasts

Gary Murray

European Journal of Biochemistry, 1987

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Characterization of glucocerebrosidase in Greek Gaucher disease patients: mutation analysis and biochemical studies

Helen Michelakakis

Journal of Inherited Metabolic Disease, 1995

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Enzyme replacement therapy in Gaucher's disease: a rapid, high-yield method for purification of glucocerebrosidase

Joseph Shiloach

Proceedings of the National Academy of Sciences, 1976

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Conditions affecting the activity of glucocerebrosidase purified fom spleens of control subjects and patients with type 1 Gaucher disease

Sonja Van Weely

Biochimica et Biophysica Acta (BBA) - Protein Structure and Molecular Enzymology, 1990

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Decreased glucocerebrosidase activity in Gaucher disease parallels quantitative enzyme loss due to abnormal interaction with TCP1 and c-Cbl

Richard Hodes

Proceedings of the National Academy of Sciences, 2010

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Relationship between Glucocerebrosidase Activity and clinical response to Enzyme Replacement Therapy in patients with Gaucher Disease type I

Vicente Giner

Basic & clinical pharmacology & toxicology, 2018

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Therapeutic response to intravenous infusions of glucocerebrosidase in a patient with Gaucher disease

Gary Murray

Proceedings of the National Academy of Sciences, 1990

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Glycolipid analysis of different tissues and cerebrospinal fluid in type II Gaucher disease

Bruno Bembi

2002

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Immunological and isoelectric focusing study of β-glucocerebrosidase from normal and Gaucher disease

Britta Hardy

Biochemical and Biophysical Research Communications, 1984

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Biochemical properties of β-glucosidase in leukocytes from patients and obligated heterozygotes for Gaucher disease carriers

Roberto Giugliani

Clinica Chimica Acta, 2005

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Enzymic diagnosis in 27 cases with Gaucher's disease

Klaus Harzer

Clinica Chimica Acta, 1980

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Synthetic substrate ß-glucosidase activity in leukocytes: A reproducible method for the identification of patients and carriers of Gaucher's disease

Cameron Clark

Clinical Genetics, 2008

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Cellular Uptake of Glucocerebrosidase in Gaucher Patients Receiving Enzyme Replacement Treatment

Matilde Merino Sanjuan

Clinical Pharmacokinetics, 2016

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Modifying exogenous glucocerebrosidase for effective replacement therapy in Gaucher disease

Gary Murray

Journal of Inherited Metabolic Disease, 1994

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Transgenic mice expressing human glucocerebrosidase variants: Utility for the study of Gaucher disease

Leanne Hein

Blood Cells, Molecules, and Diseases, 2013

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Enzyme replacement treatment in type 1 and type 3 Gaucher's disease

Bruno Bembi

The Lancet, 1994

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Mutations of glucocerebrosidase: discrimination of neurologic and non-neurologic phenotypes of Gaucher disease

Samuel Pirruccello

Proceedings of the National Academy of Sciences, 1982

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A Review of Gaucher Disease Pathophysiology, Clinical Presentation and Treatments

T Billette

International journal of molecular sciences, 2017

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Correlation between enzyme activity and substrate storage in a cell culture model system for Gaucher disease

Thomas Kolter

Journal of Inherited Metabolic Disease, 2000

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Determination of Gaucher's disease phenotypes with monoclonal antibody

Arnold Reuser

Clinica Chimica Acta, 1983

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Characterization of the c.(-203)A>G variant in the glucocerebrosidase gene and its association with phenotype in Gaucher disease

Pilar Alfonso

Clinica Chimica Acta, 2011

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Correlation among Genotype, Phenotype, and Biochemical Markers in Gaucher Disease: Implications for the Prediction of Disease Severity

Michael Fietz

Molecular Genetics and Metabolism, 2002

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Gaucher disease: N370S glucocerebrosidase gene frequency in the Portuguese population

Pedro Oliveira

Clinical Genetics, 2008

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The identification of eight novel glucocerebrosidase (GBA) mutations in patients with Gaucher disease

Brian Martin

Human Mutation, 2002

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Gaucher disease: mutation and polymorphism spectrum in the glucocerebrosidase gene (GBA)

Kathleen Hruska

Human Mutation, 2008

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Identification of a 55-bp Deletion in the Glucocerebrosidase Gene in Gaucher Disease: Phenotypic Presentation and Implications for Mutation Detection Assays

Benjamin Roa

Molecular Genetics and Metabolism, 2001

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Genotype/phenotype relationship in Gaucher disease patients. Novel mutation in glucocerebrosidase gene

Paula Arévalo

Clinical Chemistry and Laboratory Medicine (CCLM), 2020

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Glucosylsphingosine Accumulation in Mice and Patients with Type 2 Gaucher Disease Begins Early in Gestation

Brian Martin, Ellen Sidransky

Pediatric Research, 2000

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