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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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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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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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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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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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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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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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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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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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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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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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Biosynthesis of the lysosomal enzyme glucocerebrosidase

The Journal of biological chemistry, 1985-11, Vol.260 (26), p.14319-14324

1985

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

The Journal of clinical investigation, 1993-03, Vol.91 (3), p.1167-1175

1993

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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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Nucleotide sequence of cDNA containing the complete coding sequence for human lysosomal glucocerebrosidase

The Journal of biological chemistry, 1986-01, Vol.261 (1), p.50-53

1986

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Retroviral gene transfer and sustained expression of human arylsulfatase A

Gene therapy, 1996-04, Vol.3 (4), p.343

1996

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Gene Mapping and Leader Polypeptide Sequence of Human Glucocerebrosidase: Implications for Gaucher Disease

Proceedings of the National Academy of Sciences - PNAS, 1985-10, Vol.82 (20), p.7101-7105

1985

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Structure of the N-asparagine-linked oligosaccharide units of human placental beta-glucocerebrosidase

The Journal of biological chemistry, 1984-08, Vol.259 (16), p.10112-10117

1984

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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 of the glucocerebrosidase gene in mouse and human hematopoietic progenitors

Leukemia, 1995-10, Vol.9, p.S38-S42

1995

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Identification of the binding and activating sites of the sphingolipid activator protein, saposin C, with glucocerebrosidase

Protein science, 1995-04, Vol.4 (4), p.756-764

1995

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Transplantation of human hepatocytes

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

1997

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Expression of Human Lysosomal Alpha-Mannosidase Activity in Transfected Murine Cells and Human Alpha-Mannosidase Deficient Fibroblasts

Biochemical and biophysical research communications, 1996-07, Vol.224 (3), p.619-624

1996

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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

Lessons learned from the development of enzyme therapy for Gaucher disease

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

Centrifugal enhancement of retroviral mediated gene transfer

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

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

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

Risks of Gaucher's treatment

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

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

Interaction of saposins, acidic lipids, and glucosylceramidase

Biosynthesis of the lysosomal enzyme glucocerebrosidase

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

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

Nucleotide sequence of cDNA containing the complete coding sequence for human lysosomal glucocerebrosidase

Retroviral gene transfer and sustained expression of human arylsulfatase A

Gene Mapping and Leader Polypeptide Sequence of Human Glucocerebrosidase: Implications for Gaucher Disease

Structure of the N-asparagine-linked oligosaccharide units of human placental beta-glucocerebrosidase

Characterization of human glucocerebrosidase from different mutant alleles

Long-term expression of the glucocerebrosidase gene in mouse and human hematopoietic progenitors

Identification of the binding and activating sites of the sphingolipid activator protein, saposin C, with glucocerebrosidase

Transplantation of human hepatocytes

Expression of Human Lysosomal Alpha-Mannosidase Activity in Transfected Murine Cells and Human Alpha-Mannosidase Deficient Fibroblasts

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