The correction of Hunter fibroblasts by exogenous iduronate sulfate sulfatase: biochemical and ultrastructural studies.

The exogenous addition of iduronate sulfate sulfatase to cultured fibroblasts of Hunter patients resulted in a full correction of the metabolic defect as demonstrated by chemical and ultrastructural analyses. As little as 25% of the normal fibroblasts' enzyme levels were sufficient for this correction. The half-disappearance time of the internalized enzyme was 3-4 days. Prolonged incubation of corrected cells resulted in a gradual reaccumulation of mucopolysaccharides.

[1]  K. von Figura,et al.  Enzymic diagnosis of the genetic mucopolysaccharide storage disorders. , 1982, Methods in enzymology.

[2]  L. Svennerholm,et al.  Attempt at enzyme replacement by organ transplantation: renal transplantation in Gaucher disease. , 1979, Transplantation proceedings.

[3]  L. Rome,et al.  Two species of lysosomal organelles in cultured human fibroblasts , 1979, Cell.

[4]  A. Matas,et al.  Bone marrow transplantation for correction of enzyme deficiency disease. , 1979, Transplantation proceedings.

[5]  H. J. Baker,et al.  Enzyme replacement using liposome carriers in feline GM1 gangliosidosis fibroblasts , 1978, Nature.

[6]  K. von Figura,et al.  Evidence for lysosomal enzyme recognition by human fibroblasts via a phosphorylated carbohydrate moiety. , 1978, The Biochemical journal.

[7]  W. Sly,et al.  Receptor-mediated uptake of lysosomal enzymes. , 1978, Progress in clinical and biological research.

[8]  M. Cohen,et al.  Mucopolysaccharide accumulation in cultured skin fibroblasts derived from patients with mucolipidosis IV. , 1977, American journal of human genetics.

[9]  E. Neufeld,et al.  Recognition and receptor-mediated uptake of a lysosomal enzyme, α-l-iduronidase, by cultured human fibroblasts , 1977, Cell.

[10]  W. Sly,et al.  Phosphohexosyl components of a lysosomal enzyme are recognized by pinocytosis receptors on human fibroblasts. , 1977, Proceedings of the National Academy of Sciences of the United States of America.

[11]  J. Opitz,et al.  Replacement therapy for inherited enzyme deficiency: liver orthotopic transplantation in Niemann-Pick disease type A. , 1977, American journal of medical genetics.

[12]  R. Desnick,et al.  Toward enzyme therapy for lysosomal storage diseases. , 1976, Physiological reviews.

[13]  E. Neufeld,et al.  A recognition marker required for uptake of a lysosomal enzyme by cultured fibroblasts. , 1974, Biochemical and biophysical research communications.

[14]  E. Neufeld.  The biochemical basis for mucopolysaccharidoses and mucolipidoses. , 1974, Progress in medical genetics.

[15]  B. Trump.  The Network of Intracellular Membranes , 1973 .

[16]  R. Matalon,et al.  Correction of the Enzymic Defect in Cultured Fibroblasts from Patients with Fabry's Disease: Treatment with Purified α-Galactosidase from Ficin , 1973, Pediatric Research.

[17]  F. Eisenberg,et al.  The defect in the Hunter syndrome: deficiency of sulfoiduronate sulfatase. , 1973, Proceedings of the National Academy of Sciences of the United States of America.

[18]  J. Balint,et al.  Lipid accumulation and acid lipase deficiency in fibroblasts from a family with Wolman's disease, and their apparent correction in vitro. , 1972, The Journal of laboratory and clinical medicine.

[19]  E. Neufeld,et al.  A hypothesis for I-cell disease: defective hydrolases that do not enter lysosomes. , 1972, Biochemical and biophysical research communications.

[20]  A. Chrambach,et al.  The Hunter corrective factor. Purification and preliminary characterization. , 1972, The Journal of biological chemistry.

[21]  B. Weissmann,et al.  The defect in the Hurler and Scheie syndromes: deficiency of -L-iduronidase. , 1972, Proceedings of the National Academy of Sciences of the United States of America.

[22]  E. Neufeld,et al.  CORRECTIVE FACTORS FOR INBORN ERRORS OF MUCOPOLYSACCHARIDE METABOLISM , 1971, Annals of the New York Academy of Sciences.

[23]  A. Fluharty,et al.  Correction of Abnormal Cerebroside Sulfate Metabolism in Cultured Metachromatic Leukodystrophy Fibroblasts , 1971, Science.

[24]  C. W. Hall,et al.  Hurler and Hunter Syndromes: Mutual Correction of the Defect in Cultured Fibroblasts , 1968, Science.

[25]  C. W. Hall,et al.  The defect in Hurler's and Hunter's syndromes: faulty degradation of mucopolysaccharide. , 1968, Proceedings of the National Academy of Sciences of the United States of America.

[26]  T BITTER,et al.  A modified uronic acid carbazole reaction. , 1962, Analytical biochemistry.