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Enzyme replacement and enhancement therapies: lessons from lysosomal disorders

Author

Robert J. Desnick

1943 -

Edward H Schuchman

published in: Nature Reviews Genetics

date of publication: 2002-12-01

Bibliographic databases:

Cites articles 77

Date

Title

Authors

1971-06-01

Correction of abnormal cerebroside sulfate metabolism in cultured metachromatic leukodystrophy fibroblasts,Correction of Abnormal Cerebroside Sulfate Metabolism in Cultured Metachromatic Leukodystrophy Fibroblasts
1972-11-01

A hypothesis for I-cell disease: defective hydrolases that do not enter lysosomes
1973-08-01

Sanfilippo disease type B: enzyme replacement and metabolic correction in cultured fibroblasts
1973-07-01

Replacement therapy for inherited enzyme deficiency. Use of purified ceramidetrihexosidase in Fabry's disease
1974-09-01

Sandhoff disease: defective glycosaminoglycan catabolism in cultured fibroblasts and its correction by beta-N-acetylhexosaminidase
1974-11-01

Replacement therapy for inherited enzyme deficiency. Use of purified glucocerebrosidase in Gaucher's disease.
1976-01-01

Toward enzyme therapy for lysosomal storage diseases
1977-05-01

Phosphohexosyl components of a lysosomal enzyme are recognized by pinocytosis receptors on human fibroblasts
1979-10-01

Enzyme therapy in Fabry disease: differential in vivo plasma clearance and metabolic effectiveness of plasma and splenic alpha-galactosidase A isozymes
1979-06-01

Enzyme replacement in Tay-Sachs disease
1981-07-01

Characterization of a membrane-associated receptor from bovine liver that binds phosphomannosyl residues of bovine testicular beta-galactosidase
1987-05-01

Implantation of genetically engineered fibroblasts into mice: implications for gene therapy
1989

The biogenesis of lysosomes
1989-09-08

Identification of the cystic fibrosis gene: genetic analysis
1990-06-01

Lysosomal enzyme targeting
1990-03-01

Therapeutic response to intravenous infusions of glucocerebrosidase in a patient with Gaucher disease
1991-05-01

Replacement therapy for inherited enzyme deficiency--macrophage-targeted glucocerebrosidase for Gaucher's disease
1992-01-01

Structure and function of the mannose 6-phosphate/insulinlike growth factor II receptors
1992-03-01

Neuronal lysosomal enzyme replacement using fragment C of tetanus toxin
1992-12

Overexpression of human alpha-galactosidase A results in its intracellular aggregation, crystallization in lysosomes, and selective secretion
1992-06-01

Animal model of Gaucher's disease from targeted disruption of the mouse glucocerebrosidase gene
1992-10-01

Gaucher disease in the neonate: a distinct Gaucher phenotype is analogous to a mouse model created by targeted disruption of the glucocerebrosidase gene
1993-11-01

Mouse models of Niemann-Pick disease: mutation analysis and chromosomal mapping rule out the type A and B forms
1995-07-01

Mannose 6-phosphate receptors in sorting and transport of lysosomal enzymes
1995-10-01

Mouse models of Tay-Sachs and Sandhoff diseases differ in neurologic phenotype and ganglioside metabolism
1995-06-01

Acid sphingomyelinase-deficient mice mimic the neurovisceral form of human lysosomal storage disease (Niemann-Pick disease)
1995-09-01

Galactose stabilizes various missense mutants of alpha-galactosidase in Fabry disease
1995-01-01

Enzyme therapy in type 1 Gaucher disease: comparative efficacy of mannose-terminated glucocerebrosidase from natural and recombinant sources
1996-05-01

Bioproduction of human enzymes in transgenic tobacco
1996-12-01

Phenotype of arylsulfatase A-deficient mice: relationship to human metachromatic leukodystrophy
1996-08-01

Targeted disruption of the arylsulfatase B gene results in mice resembling the phenotype of mucopolysaccharidosis VI
1996-01-01

Glycerol reverses the misfolding phenotype of the most common cystic fibrosis mutation.

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