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Long-term expression and transfer of arylsulfatase A into brain of arylsulfatase A-deficient mice transplanted with bone marrow expressing the arylsulfatase A cDNA from a retroviral vector

Gene therapy, 2000-07, Vol.7 (14), p.1250-1257

2000

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Risks of Gaucher's treatment

The Lancet (British edition), 2000-10, Vol.356 (9238), p.1353-1354

Barranger, John A

2000

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Guidance on the use of miglustat for treating patients with type 1 Gaucher disease

American journal of hematology, 2005-11, Vol.80 (3), p.223-229

2005

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The effect of cationic liposome pretreatment and centrifugation on retrovirus-mediated gene transfer

Gene therapy, 1997-12, Vol.4 (12), p.1379-1386

1997

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Radiological Evidence of Early Cerebral Microvascular Disease in Young Children with Fabry Disease

The Journal of pediatrics, 2005-07, Vol.147 (1), p.102-105

2005

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Correlation of surrogate markers of Gaucher disease. Implications for long-term follow up of enzyme replacement therapy

Clinica chimica acta, 2004-06, Vol.344 (1), p.101-107

2004

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Clinical trial to assess the safety, feasibility, and efficacy of transferring a potentially anti-arthritic cytokine gene to human joints with rheumatoid arthritis

Human gene therapy, 1996-06, Vol.7 (10), p.1261-1280

1996

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Lessons learned from the development of enzyme therapy for Gaucher disease

Journal of inherited metabolic disease, 2001-01, Vol.24 (S2), p.89-96

2001

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Demonstration of Feasibility of In Vivo Gene Therapy for Gaucher Disease Using a Chemically Induced Mouse Model

Molecular therapy, 2002-08, Vol.6 (2), p.179-189

2002

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Transplantation of human hepatocytes

Transplantation proceedings, 1997-06, Vol.29 (4), p.2103-2106

1997

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Centrifugal enhancement of retroviral mediated gene transfer

Journal of virological methods, 1995-08, Vol.54 (2-3), p.131-143

1995

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Identification of the neurotrophic factor sequence of prosaposin

The FASEB journal, 1995-05, Vol.9 (8), p.681-685

1995

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Surrogate markers for lysosomal storage

Blood, 2004-01, Vol.103 (1), p.2-3

2004

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A mutation in the human glucocerebrosidase gene in neuronopathic Gaucher's disease

The New England journal of medicine, 1987-03, Vol.316 (10), p.570-575

1987

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Efficient Transfer and Sustained High Expression of the Human Glucocerebrosidase Gene in Mice and their Functional Macrophages Following Transplantation of Bone Marrow Transduced by a Retroviral Vector

Proceedings of the National Academy of Sciences - PNAS, 1992-12, Vol.89 (23), p.11332-11336

1992

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Genetic Heterogeneity in Type 1 Gaucher Disease: Multiple Genotypes in Ashkenazic and Non-Ashkenazic Individuals

Proceedings of the National Academy of Sciences - PNAS, 1988-04, Vol.85 (7), p.2349-2352

1988

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Gene transfer approaches to the lysosomal storage disorders

Neurochemical research, 1999-04, Vol.24 (4), p.601-615

1999

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Detection and isolation of gene-corrected cells in Gaucher disease via a fluorescence-activated cell sorter assay for lysosomal glucocerebrosidase activity

Blood, 1997-05, Vol.89 (9), p.3412-3420

1997

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Hematopoietic stem cell gene transfer

Gene therapy, 1996-05, Vol.3 (5), p.379

Barranger, J A

1996

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Retrovirus-mediated transfer of human α-galactosidase A gene to human CD34+ hematopoietic progenitor cells

Human gene therapy, 1999-12, Vol.10 (18), p.2881-2889

1999

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Long-term expression and secretion of human glucocerebrosidase by primary murine and human myoblasts and differentiated myotubes

Journal of molecular medicine (Berlin, Germany), 1998-10, Vol.76 (11), p.773-781

1998

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Characterization of human glucocerebrosidase from different mutant alleles

The Journal of biological chemistry, 1991-02, Vol.266 (6), p.3661-3667

1991

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Long-term expression, systemic delivery, and macrophage uptake of recombinant human glucocerebrosidase in mice transplanted with genetically modified primary myoblasts

Human gene therapy, 1998-11, Vol.9 (16), p.2375-2384

1998

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Interaction of saposins, acidic lipids, and glucosylceramidase

The Journal of biological chemistry, 1990-02, Vol.265 (4), p.1933-1937

1990

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Fabry disease in genetic counselling practice: recommendations of the National Society of Genetic Counsellors

Acta Paediatrica, 2002-11, Vol.91 (s439), p.122-122

2002

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Long-term expression and transfer of arylsulfatase A into brain of arylsulfatase A-deficient mice transplanted with bone marrow expressing the arylsulfatase A cDNA from a retroviral vector

Risks of Gaucher's treatment

Guidance on the use of miglustat for treating patients with type 1 Gaucher disease

The effect of cationic liposome pretreatment and centrifugation on retrovirus-mediated gene transfer

Radiological Evidence of Early Cerebral Microvascular Disease in Young Children with Fabry Disease

Correlation of surrogate markers of Gaucher disease. Implications for long-term follow up of enzyme replacement therapy

Clinical trial to assess the safety, feasibility, and efficacy of transferring a potentially anti-arthritic cytokine gene to human joints with rheumatoid arthritis

Lessons learned from the development of enzyme therapy for Gaucher disease

Demonstration of Feasibility of In Vivo Gene Therapy for Gaucher Disease Using a Chemically Induced Mouse Model

Transplantation of human hepatocytes

Centrifugal enhancement of retroviral mediated gene transfer

Identification of the neurotrophic factor sequence of prosaposin

Surrogate markers for lysosomal storage

A mutation in the human glucocerebrosidase gene in neuronopathic Gaucher's disease

Efficient Transfer and Sustained High Expression of the Human Glucocerebrosidase Gene in Mice and their Functional Macrophages Following Transplantation of Bone Marrow Transduced by a Retroviral Vector

Genetic Heterogeneity in Type 1 Gaucher Disease: Multiple Genotypes in Ashkenazic and Non-Ashkenazic Individuals

Gene transfer approaches to the lysosomal storage disorders

Detection and isolation of gene-corrected cells in Gaucher disease via a fluorescence-activated cell sorter assay for lysosomal glucocerebrosidase activity

Hematopoietic stem cell gene transfer

Retrovirus-mediated transfer of human α-galactosidase A gene to human CD34+ hematopoietic progenitor cells

Long-term expression and secretion of human glucocerebrosidase by primary murine and human myoblasts and differentiated myotubes

Characterization of human glucocerebrosidase from different mutant alleles

Long-term expression, systemic delivery, and macrophage uptake of recombinant human glucocerebrosidase in mice transplanted with genetically modified primary myoblasts

Interaction of saposins, acidic lipids, and glucosylceramidase

Fabry disease in genetic counselling practice: recommendations of the National Society of Genetic Counsellors

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