Correction of human mucopolysaccharidosis type-VI fibroblasts with recombinant N-acetylgalactosamine-4-sulphatase
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چکیده
منابع مشابه
Correction of human mucopolysaccharidosis type-VI fibroblasts with recombinant N-acetylgalactosamine-4-sulphatase.
A full-length human N-acetylgalactosamine-4-sulphatase (4-sulphatase) cDNA clone was constructed and expressed in CHO-DK1 cells under the transcriptional control of the Rous sarcoma virus long terminal repeat. A clonal cell line expressing high activities of human 4-sulphatase was isolated. The maturation and processing of the human enzyme in this transfected CHO cell line showed it to be ident...
متن کاملN-acetylgalactosamine 4-sulphatase protein
Doug A. BROOKS,*: Daniel A. ROBERTSON,* Colleen BINDLOSS,* Tom LITJENS,* Don S. ANSON,* Christoph PETERS,t C. Phillip MORRIS* and John J. HOPWOOD* *Lysosomal Diseases Research Unit, Department of Chemical Pathology, Centre for Medical Genetics, The Women's and Children's Hospital, 72 King William Road, North Adelaide, South Australia, 5006, Australia and tBiochemie 11, Universitgt Gottingen, Go...
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BACKGROUND AND METHODS: Growth failure is characteristic of untreated mucopolysaccharidosis type VI (MPS VI: Maroteaux-Lamy syndrome). Growth was studied in fifty-six MPS VI patients (5 to 29 years old) prior to and for up to 240 weeks of weekly infusions of recombinant human arylsulfatase B (rhASB) at 1 mg/kg during Phase 1/2, Phase 2, Phase 3 or Phase 3 Extension clinical trials. Height, weig...
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Mucopolysaccharidosis type VI (MPS VI) is an autosomal recessive disease caused by a deficiency of N-acetylgalactosamine 4-sulfatase (4S) leading to the lysosomal accumulation and urinary excretion of dermatan sulfate. MPS VI has also been described in the Siamese cat. As an initial step toward enzyme replacement therapy with recombinant feline 4S (rf4S) in MPS VI cats, the feline 4S cDNA was i...
متن کاملRecombinant human iduronate-2-sulphatase: correction of mucopolysaccharidosis-type II fibroblasts and characterization of the purified enzyme.
Mucopolysaccharidosis type II (MPS II, Hunter syndrome) is an X-chromosome-linked recessive lysosomal storage disorder that results from a deficiency of iduronate-2-sulphatase (12S). Patients with MPS II store and excrete large amounts of partially degraded heparan sulphate and dermatan sulphate. In order to evaluate enzyme-replacement therapy for MPS II we have expressed a chimaeric I2S cDNA i...
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ژورنال
عنوان ژورنال: Biochemical Journal
سال: 1992
ISSN: 0264-6021,1470-8728
DOI: 10.1042/bj2840789